36 Burst results for "cystic fibrosis"
Fresh update on "cystic fibrosis" discussed on The Joys Of Binge Reading: The Best in Mystery, Romance and Historicals
"Go find out from being there I mean Jackie is a big swimmer and I love swimming as well. So I could really see what she loved. Being on the island, the waterways absolutely crystal clear, and you could swim quite easily from one day to the next around there. This is really good roots that you could swim. I could see why should I mean? It's just stunning landscape, but I serve in the island we were staying on, which is called lift Carter wondered ratliff Carter town and I came across an old man who had a Newseum, the phonograph in his front room. He just had to sign up and he had a collection of of rare recordings and so forth and he told me about a time when Maria Aristotle came for dinner in Lefkada and she stood up and gave an impromptu concerts and he'd been there you had a couple of photographs. He said it was one of the fondest memories of his life. How amazed so little things that are. In it didn't make its way into the novel but. It was just lovely to he their local heroes. There's there's a statue of Aristotle along the either an lots of restaurants have a dish that was said to be his favourite which is. Enormous cholesterol heavy thing of Eland pork and cheese tomato. An I ordered at one nine to photograph of it. ICON actually eat that. Sit Greasy. Favorite meal. A. Yeah. No I had a lawsuit fund researching this one for sure. This ninety mation sympathy I took Wanda. Do you feel more sympathy for one or the other entity debt failing changes. He wrote the book I I was. I felt the Maria needed reassessment more than Jackie did because. So many biographies carry on this image of difficult. Gutman. Hydro No I. Think it's a feminist thing. But when somebody calls a woman difficult and reasons the reasons for for example that she. Some perfectionists about her Kroft if she had to concerts, she would ask the orchestra to stay and keep her seat until she was absolutely. Ready And maybe that major unpopular dramas with other opera singers less Scala around the world. That were blown up by the press office to get people to come to concerts. It's just it was all manufactured mostly. The other thing was Maria is that her own mother spoke out against her she gave. An interview to a Time magazine journalist Saint had difficult. Maria was a daughter and and then she wrote a memoir saying that bizarrely Maria has been in a car accident at the age of five and it made her really twisted pitcher character which there's no evidence for tool and I thought oh my gosh with mothers. Let you know who needs enemies. It's a horribly she really goes short store with families. So the more I read the more. I was disbelieving these people say that she was a difficult character while I noticed reading between the lines was that she had a large extremely loyal of friends and that made me lonely Kurt Yeah Yeah. That was the first step you know I really felt that it was getting to know her Jackie. Kennedy I came with far more could preconceived ideas so I wasn't assessing her quite so much for myself. I wanted to answer the questions Why did she stay with a man who was unfaithful to her on an industrial scale? I'm talking about John, f.? Kennedy I'm. Not. Just. All over the you know he was just sleeping with empathy. One question. But also, why did she marry Onassis when she had so many other choices there. What was she thinking of you? She didn't have the incumbent with him. So I just kept reading for me my own patron till. I thought I could understand it now I'm not saying this is the truth Iverson. But I try to myself into their shoes and see what it must've felt be them and come up with version of them feels emotionally true to me. So I guess there's a bit of my thinking in there as well. I'm definitely adult saying I've got the right answer. I think one of another of the Jackie books that I read actually has even hinted that that. Sister League may have even Deli was. K WHO Was Janke suspect yard the much lower aside really was. Well Lee Lee's first husband Michael Canfield claims that he overheard her having sex with JFK the next room so But. Perhaps he was bitter because she divorced him. On Friends of Jackie's who Jackie said that she knew about the time and just with her icq decided not. Rock the boat. Or decisions. In It is the joys of binge reading. So we do want to quickly check back with you on what you might be reading now and anything that you've got to wait a mean for us what do you? What are you? What are you into at the moment? I've actually been reading loads of historical fiction this summer because I'm a judge of the historical Writers Association. Novel Competition. Switch has been wonderful I've been forced to read. Ancient. Roman Greek and medieval and all kinds of historical fiction that I wouldn't normally pick up myself and yet discovered some wonderful books. One of them are really lights mentioned is, and by the way I'm not the judges have chosen the winner yet. But Yeah, one of my favorites is by Sarah and it's called coming up for air. She's a Canadian with right never heard of before. Novels. Set in three different time periods with different characters, different countries, which can be a difficult thing to do but I was quickly absorbed in each the stories. There's one in eighteen nineties Paris as a young girl is about to jump off a bridge into the San at night and Q. There's toymaker in Norway in the nineteen fifties giving the island's a very tough lifestyle very harsh, and then there's A. Girl in nineteen, thousand, nine, hundred, ninety s Ottawa in Canada because cystic fibrosis and every morning has to cure her nuns in order to stay alive and it's about breathing and swimming and drowning, and that fine line between being alive and not being live anymore is really beautifully written book and the three clots. which are equally absorbing come together in such a clever way I, can't tell you how they come together. So Clever I. Love. Its right. That's wonderful. Now, you mentioned swimming number of times. When we took last time, you can how you swim year round in London and pool in. The pack near you had been able to continue with that. This is GonNa make sense foil. The worst thing about lockdown for me was that my pond closed for four months while they figured out ways to make it carbon safe it real July an upgrade there every day moment. We have to book a times thoughts that. Go in a very organized way. So. That spontaneity account just finish writing a chapter of the rules. But yet, no, I.
Never Be Royals
"It has been the habit of kings throughout the world to hire tasters to test their food on the off chance. Some oppressed masses poisoned it. But Henry, the eighth cast, a wider net with his paranoia. He wanted to be certain. No one was going to poison him transdermal either meaning through the skin. He ordered that every morning, the servant, the WHO changed the kings sheets had to kiss every part of the sheets, pillows and blankets they had touched to prove they hadn't smeared poison on them. They also had to test for poison on the cushion of his son. Edwards Chamber Pot. Though the historic record doesn't say how? My Name's Moxy and this is your brain on facts. The term mad king re entered the common lexicon a few years back. Thanks to George R. Martin's Song of ice and fire books. We'd have more than enough examples from Europe alone to choose from. George. The third born in Seventeen thirty eight was the English king who lost the American colonies. Though anti-monarchists would record stories of bizarre behavior like George Mistaking and trees for Frederick the Great? The king really did have mental health problems that manifested themselves in different periods of his life. During these times, he suffered from insomnia and talked incessant nonsense for hours. It was not uncommon for a single sentence to contain four hundred words. It has long been suspected that King George suffered from Porfirio. A genetic metabolic disorder that causes depression hallucinations, constipation, purple urine, and severe abdominal pain. However, as will come up frequently today new evidence in theories undermined that original thinking. One of the medicines that king was treated with was Gentian. This plant with its deep blue flowers is still used today as a mild tonic that may turn the urine blue. His incessant liquidity as it was called and his habit of talking until foam ran from his mouth are features that can be seen today in patients with extreme cases of mania from psychiatric illnesses such as bipolar disorder. Besides benign sounding herbal treatments, King George would be restrained in a chair with iron straps for hours. He was also bled forced to vomit and starved suffering under the humour old or four humors school, of medicine. A recent study based on the examination of King. George's hair shows high levels of arsenic administered to him as part of the cure, but would have served to only worsen his symptoms. In the last ten years of his life, his son and Heir George, the fourth served as regent. Fans of the show blackadder will remember George the fourth brilliantly portrayed by Hugh Laurie opposite the Tischler, Rowan Atkinson character. A somewhat annoying little side note when the play the madness of George the third was made into a film. In nineteen, ninety four, the title was changed to the madness of King George. Why for fear that American audiences would think it was a sequel and wouldn't go to see it because they hadn't seen the first two. When your business is running countries and even whole empires you WanNa keep that in the family and the best way to ensure that is to make sure everybody marries somebody there already related to. When you say it like that, it doesn't sound like such a good plan. But that was how royal houses conducted themselves for centuries to ensure they retained their fortunes in the days before even a basic understanding of genetics. When close relatives reproduce, it increases their offspring's chances of being affected by. Recessive traits for all kinds of physical and cognitive disabilities, including things like hemophilia and cystic fibrosis, as well as deformities like the Habsburg jaw. These incestuous pairings also run a greater risk of reduced fertility higher infant mortality, congenital birth defects, certain kinds of cancer suppressed immune systems, and overall smaller adult size. A condition referred to as pedigree collapse. Some royal families kept things closer-knit than others. Maria I of Portugal married her father's younger brother Pedro when she was twenty six and he was forty three. Their son and Heir Joseph married his aunt Maria Sister Benedetta. Therefore, Pedro's daughter-in-law sister
"cystic fibrosis" Discussed on Newsradio 950 WWJ
"With cystic fibrosis and I realized I could help others on behalf of all the people we've helped thank you W. W. JT is time to forty eight traffic and weather together on the eight says we hit the roads and start a Macomb county with Tracy and we're watching here through the two filet GM Kansas in an accident they are along I. ninety four in Clinton township and this traffic update I should say sponsored by Greek town casino so here I. ninety four westbound at Harper a crash blocking the right lane and shoulder this affects the exit to Harper is well traffic slower from Schiff wrote better news seventy five north there eight mile road that accident out of the way looks pretty good for now I. ninety four in fifty nine and fifty three and no problems along six ninety six through warranty and the south field the nineteenth annual D. three Greek independence day parade celebrating Greek American culture is set for this March twenty ninth at three PM in historic Greektown Detroit after the parade stop in for fine food and great gaming at Greektown hotel and casino must be twenty one to gamble on the adult on motor group twenty four hour traffic center Tracy because go W. W. J. newsradio nine fifty accu weather's John fear it now excuse me dean devore has our forecast from the family heating cooling and electrical weather Saturday in varying amounts of clouds up to fifty two this afternoon some rounds of showers moved through tonight maybe a downpour and rubble south of the city breezy colder towards daybreak as we get it down about thirty seven and then windy and chilly tomorrow as sunshine mixes some clouds mid forties at best on the thermometer but it'll.
"cystic fibrosis" Discussed on Columbus Concerns
"This is Columbus concerns a public affairs presentation of North American Broadcasting. Here's your host Mark News. Good morning and welcome to the program. Mark New Sierra. And we're so glad that you joined us this morning on Columbus concerns going to talk with a couple of friends of ours. Who have been on the program. Before from the CYSTIC Fibrosis Foundation. We have Jennifer Hughes the Development Manager and aid the Angelo the development director. And thanks for coming by today. Now I understand that you have A lot of events coming up In the spring a lot of fundraising events. A lot of fun. Things that you guys do Why don't you start off telling me about the first one coming up here in February soon? Yeah absolutely it's our nausea gala event. And this is coming. Up on February twenty-second it'll be at five thirty PM at the Ivory Room Down Amir Nova a very nice place down there. this is kind of a a black tie affair It is put on by the CYSTIC fibrosis foundation in support of our patients of what makes nausea released special. Is We have something. We work with Camera Mitchell on where we get six of camera. Mitchell's top chefs to make an appetizer. That would be beneficial for. Cf patient so we use ingredients that would be beneficial for them as our guests come in. They try all the appetizers and vote on their favourite one. So that Kinda kicks off the night with our little chef's tasting outside of that. We have a full dinner wonderful presentation and of course band kicks in. There's lots of dancing and music and this year. We're even going to have popcorn bar so it's going to be a lot of fun and all in another benefit for cystic fibrosis. Goes on like a lot of fun and How do people get more information about it? Absolutely you can go online at events dot. Cf Dot org forward slash nausea which is an S. H. And you can get tickets online right there and Is there a deadline for registration We need to have everyone's registrations and by next Thursday so if you want to come to the event Make sure you get online right away or you can also call the office at six one. Four eight nine zero six five six five asked for me. Amy and be glad to help you out now. I hope everybody brings her their phone or their hammer. Because that is one of the best places in Columbus to see a skyline view. Get great pictures. Absolutely the ivory absolutely stunning on the view is amazing Especially as the sun goes down at night and our cocktail hours going on. It's it's really quite stunning. It's a beautiful place to be. It'll be a wonderful night. Jennifer told me about the event coming up in March so the event coming up in March is called. The Columbus brewers ball and it is a celebration of our finest campaign and what the finest campaign is is a Sixteen Week. Fundraising campaign to nominate one of Columbus is finest young professionals and these Honorees are nominated typically in October and then we have a selection and a announcement in November and they spend the next sixteen weeks fundraising for the CYSTIC fibrosis foundation. Essentially dollars equal votes. So when we celebrate at the brewers ball this year that is when we will be recognizing our honorees and announcing the winner will receive a great prize as well as their head shot on a billboard down in grandview for a month than some other things as well. The brewers ball itself is going to be view. Columbus downtown on March fifth at six thirty PM and this event is a tasting and by the bite type thing with a lot of our local breweries and local restaurants as well as a live band and a DJ and photo booth and Silent Auction and so much more. It's going to be a really fun. High Energy Event and it is something that we're kind of expanding and turning into more of a community event wrapped around a really great cause another great venue downtown where people can come out Tell me a little bit about the tickets for that. How do people get involved so to get tickets? For the brewers Vol you can visit the website at. Www dot finest dot cf dot org forward. Slash Columbus brewers ball and there will also be tickets available at the door for this event so take a peek at the website. See who all of our brews and food spirit vendors are going to be joining us this year. And I think everybody will be pleasantly surprised on the program today Jennifer Hughes. Abd Angelo from the CYSTIC Fibrosis Foundation. Central Ohio chapter and filling us in on some of the fun events that they have coming up this spring and They've talked about one in February which you need to get on right away and another one in March and of course. Your spring culminates in May with the great strides walk. Amy Tell me a little bit about The Walk and how people get involved in that absolutely great strides is our biggest fundraiser. And it's something that's actually done all across the country in May In support of cystic fibrosis. Our walk the main walk will be in Columbus on May third down at McPherson Commons. Park the walk itself will begin around noon But YOU WANNA come down for that you can get registered online. Cff Dot org forward slash great strides? You can just sign up your team or you can come just as an individual walker or if you can't make it down there for that day you can actually just be a virtual walker and just support the calls on that day as well. Great Strides is a lot of fun for our families. It's really important to them they Oftentimes CF is a is a disease where one person with cf can't be near person. Another person with cf but at great strides gives them the opportunity to be together. Because they're outside so it's a pretty important and very special day for our families lots to do out there. We've got stilt walkers and princesses walking around and lots of food and it's just a wonderful day for everyone and Jennifer these types of events are always great not only but she meant for individuals or maybe a family group for corporate groups. It's great it's like a team building effort. It's absolutely an opportunity for team building. And we do have many corporate teams that join us from for great strides and they may have somebody who's personally connected to the organization or they might not just might find it to be a wonderful organization to raise awareness for and to support research for our patients so they'll fundraise and come out with a large group walk and enjoy all the festivities and it's quite impressive amy. Do you have a website for the great strides walk absolutely at? Www dot co dot org forward slash great strides. That one's easy easy one absolutely and the phone number two for the organization if somebody wants to call and have a question if or maybe they're not as good with a computer. If anybody has any questions about any of our events the chapter you can always reach out to our office at six one. Four eight nine zero six five six five and we'll give the website addresses in the phone number again before the programs over. If you want to grab a pen and get this information. Of course you can always just google it as well and anytime Jennifer US and Amy Dangelo here from the cystic fibrosis foundation and Amy Ah I know that Your Foundation is very active. You do a lot of the fundraising and stuff a tell people where the money goes from these fundraisers all the money that we raised goes directly into research for CF I'm sure I don't know if your population is her. But we just recently released a drug called trae Kafka that is helping close to ninety percent of our patient population. So what's interesting about that? And what's wonderful to see is all of these fundraising efforts that we've done all of the events that Jen and I put on and all the ones that are across the country all filter into making things like that drug happen and that's really very special very special to our patients but the piece of that is trike. Afteh only affects about ninety percent. They're still ten percent of our population. That don't have anything that can help them and that's where we are still pushing instill fighting and still fundraising to make sure that those patients get the medicine that they need to be better. So that's where all the money goes it's going to. S- to help those families into eventually make cf Stanford cure found Jennifer. Tell us a little bit about For those who don't know. What exactly is cystic fibrosis? And how many people does it impact? Cystic fibrosis is a genetic disease. That affects our patient's lungs.
An Interview with Jockey Daniel Centeno
"He's won nearly twenty nine hundred races here in the United States and nearly nine hundred more in his native Venezuela. Many of those wins have come Tampa Bay downs. Where Jockey Daniel? Centennial was in search of his seventh riding title even earned his first ride in a triple crown race. The preakness where we finished eleventh on it always mining all jockeys have obstacles to overcome. That's obviously the nature of the business. But one year ago Daniel Centennial had an even tougher burden to bear in January of two thousand. Nineteen centennial's longtime partner Ashley. George died after a nearly lifelong battle with cystic fibrosis roses. She left behind their daughter. Jasmine who's now in seventh grade. It's tough enough being a single dad and oh by the way Santana also has a twenty year old son son in college but it's pretty tough to work from home when you do what he does. So let's find out how he makes it all work as we welcome for the first time here to win the gate jockey. Daniel Centeno already. A handful of wins for you at the Tampa Bay. Meet a place where you've had quite a bit of success over the years more so than in Maryland where you ridden over the summer. What's been the key to your success in Tampa I dunno being here for light Fifteen sixteen years now for my first time and I I talk like it. I love the weather. I'm never been relieved. Grind with the cold weather. I never try to stay up on the winning thing and I did year by year. You know writing better courses that train and I got to my support here at the beginning from gaming as Kathy O'CONNELL and ride them for their like. WHO So? Maybe you know. Open a lot of a lot more awesome around the track and you know all my area that had been working for Greg to you split you. Year between Laurel Park in Maryland and Tampa. But now I know we're not going to see you what aqueduct in the winter based on what you just said but what made you choose those tracks supposed to say New York or California well because People that are right in for here in Tampa wants to go to Laurel Delaware and the year see bars. So that's like my shadow. God Nora Bill and it's pretty time so I'm really basically driving them. Everywhere are but We're doing good and a lot of oxygen around jockeys are said to have particular writing styles go for example Paco. Lopez takes horses away from the gate aggressively. Everybody knows that likes to go to the early lead. Pat Day was known as wait all day coming from behind. Nine Calvin Burrell is Calvin Bo Rail. You know where he's taking is horses. What do you consider to be your style? Well I I would say eighty two. I like to be more delay but You know it depends on what kind of house I've gone awry but I would like to be close to my thousand dollars outbreak throats to the Leo to pay Being the lead. But it's a defendant to pry. Sometimes you gotTa Jenny a little bit your style to fit all on the horses you know and then workout good too but I'm I feel more comfortable. Really close to the lay or something. That'd be proponent department of your nearly two thousand nine hundred career wins. You've won five grade three races in your career and a single grade to aboard ring weekend in the two thousand fourteen Tampa Bay Derby. You weren't a ride in the preakness as well in two thousand nineteen. What do those kinds of races mean to you? They they mean a lot for me. You know because I can help your mom. I Don t still down so I'd be doing building my curry of keying in this country like little by little step-by-step working hard right in the for different track The Great State for me the main a lot for me you know like I were so hot. And it's like a bay off for my work and I'm never give up and try you know especially luckier when I wrote a pregnant of like a drink of through. You know the the big horse and it would broaden your really good him. Kelly Rowland the ordinary. They give me the opportunity right. Trading is what so I drink too for me. Was it hard not to look around and just take in the atmosphere and instead focus on what you were doing. How hard list to do? Yeah well I come from Venezuela complete different compare here and then you really have to focus at work workout every day so you can get your business and you know doing your job and you know made me thanks you know and then try to cloud what every day. How hard was it making the adjustment from Venezuela coming here as a young man not sure of the language and not knowing what's at your prospects would be? What was that adjustment like well? There's a big chance you know especially with the with the language that you have to really talk to do every body and understand what they wanna tell you frighteners on there. When I was in Minnesota last dot doing English class and private I went to high school and in a private school so when I came back here I can't hear one time? Nineteen Ninety six. So I didn't really good because language I think more mar and then went back to Venezuela so I wanNA come back yet again and and do the same. That's what I do. Day lift classes private and Eleven when again by Two Thousand and three door. Hi You I was big little be I understand a little bit and then I got it your breakfast every day. You're talking every day so make as you Jackie Daniels. Centeno joins us here on the gate. It's been a year or so now. I belief since your partner Ashley George passed away from cystic fibrosis. What was it like losing her? And what's alive. You know US work together for the book thing and we went to a lot of work on every time Fago. What's getting worse and especially my daughter Yachtsman but I'm blessed my daughter's so throng which Banja La doing amazing and Making it every day you gotta be strong for my daughter and then their best for her. Now I believe when you have cystic fibrosis yes you have it almost your entire life so you must have known when you met her that she had this condition. Is that right. Yeah Yeah Yeah keep telling me from the beginning to doing doing okay. But in the meantime wanting her lungs would getting worse and worse than that was boring. They're are there only chantey how they would've transplant. And the medical cameras are not that both longtime plans doing quake and one day. Keep them back home for Halloween. And she that stroke and she was to call my mom to wake like Lamb and never for memory to talk in everything but no memory and keep going. I asked me and see what's in the two years and and doctors say to her body was rejecting everything again and then get get Her body stop working. Did you say she was in a hospital for two years. was that kind of in and out or was she in a hospital all the time time for two years to two years. Happy because you know she can be herself grandma what they get her. I was flying back and forth some time with me to see her but kick No recognize anybody know memories your longtime memories. The more you so I went to Work Yom okay and too much medication So it was really good time for my daughter and either adopt nine so oh my goodness now. It's one thing to being a dad but it's another thing when those kids are both teenagers because says many of our listeners know teen is four letter word I know I have a teenager and yours are on the opposite end of the teenage spectrums. Just when when you finish the teenage thing it all starts over again. How have you been able to manage all of that and your job well? And that's another the team did it. Don't nobody could All I think I'm blessed and my father and me to do everything so and then report report anything. I wasn't never pectin something like that but I was thinking
"cystic fibrosis" Discussed on The World and Everything In It
"Treatment cystic fibrosis or. CF is a rare genetic disease that affects about seventy five thousand people around the world. The disease leads to chronic lung infections and long-term damage and Short life the life expectancy for someone with. CF is about thirty seven years. There's but just last month. A new treatment hit the market and it is a game changer. World Radios Anna. Joe Hansen has our story everyday. Jackie Miller Miller has a routine. Oh Yeah Oh yeah. There's definitely a time. Consuming regimen that regimen includes using a nebulizer two or three times today. Handfuls of pills regular injections enzymes antibiotics insulin. A vibrating vest to help break up mucus in her lungs. If she gets a cold she could would end up in the hospital. Jackie Miller has cystic fibrosis ends and genetic disease that affects anywhere in the body that produces mucus which turns out could be on the body. There's a certain protein. That's in charge of getting salt through your cell membranes if you have. CF You have a genetic mutation that botches is that specific protein. It can't do its job. So salt stays trapped inside the cell and so it draws all the moisture in the cells and because that you get really thick and sticky Nukus that thick and sticky mucus builds up and affects the liver kidneys. Intestines the pancreas and end the lungs. It leads to lung infections lung damage and lung failure. It wasn't ghastly disease to be very honest with you. That's Bonnie Ramsey. She's a pulmonologist and she spent a lot of her career figuring out ways to treat. Cf How could you develop treatments. That wouldn't maybe not correct the underlying defect but could help with the secondary consequences. So that even though we it didn't have a quote cure the quality and duration of life was improving steadily but instead of just treating the symptoms tmz researchers wanted to fix the root of the problem the actual botched protein and that is where traffic comes in is a combination of three uh-huh medications. That's Brian O'Sullivan. He's a pediatric pulmonologist at Dartmouth. Two of these medicines work on helping the protein fold improperly. So he's talking about the protein in charge of moving salt out of cells. O'Sullivan says that protein is like a gate in offense. The cows in one pastor want to get to the other side but the gates broken so one medicine fixes the gate another moves it into the right position then. The third medication comes into play third. It is the WD forty of The sell it up the gate so it can swing wide open and let the cows through or in this case. Let let the salt out of the cell. It's the first time researchers have tried combining all three pieces. They tested it into clinical trials on more than five hundred patients. Jackie Miller was one of them. She took the medication for thirty days. It was a double blind study so she didn't know if she was getting the active drug or a placebo but she could could feel the difference almost instantly and she knew she had the real thing. Like if I laugh really hard at anything it just sends me into a coughing fit so that was one thing that to change that I noticed immediately as I was laughing and then I just stopped and so that was the DR and then When I take a deep breath I hear and I feel crackles my Hsa so really early on with the drug? I would notice the crackles. We're not there Bryan. O'Sullivan says the truck after trials were enormously successful. They're breathing anything test. Went up by ten percent which is a huge amount their need for hospitalization or added antibiotic therapy went down by about seventy seventy percent compared to the group that didn't get the trike Afteh so there was a huge improvement is expected to help ninety percent of people with cf researchers are still working to find a treatment for the other ten percent but this is a huge step forward. Bonnie Ramsey has been part of the research effort for years she recalls was a moment early on in the process and I looked at the data and I thought this can't be real. You can't have this kind. Had a change made it happens almost immediately and I remember thinking this will be life changing the FDA approved to try capita in late. October Bryan. O'Sullivan says he's already writing prescriptions for it but people who need trifecta also need some really good insurance estimates put the cost of the drug somewhere north of three hundred thousand dollars per year but because not many people have the condition. Not many will need the medication. No Sullivan expects most health. Insurers will be willing to cover the cost and even with the Steep Price Tag. Jackie Miller says it's worth it before she wouldn't let herself think about the future. Now that's changing. I feel like I've been given permission to think more about like if my kids ever have kids and I have a chance to grandma so it's just been a really really sweet gift reporting for World Radio. I'm Ana Joe Hanson. Manson all right. This is a great story. You're GONNA love this. An intruder in Rochester. New York picked the wrong wrong house to break into inside that wrong house was Willie Murphy. Eighty two years old. A grandma she was home alone when a guy pounded on her door claiming to need an ambulance of course he didn't really need it but shortly after he kicked her door down he would not give you the details. Just a minute. You see Murphy was not the easy target target. Maybe he thought she was as she explained W. H. A. MTV. I'm alone and I'm old but guess what I'm tough. Guess what. AWW tough doesn't even capture it. It is entirely too small. A word for the toughness that is Willie Murphy. She is a bodybuilder. While she's only five feet tall weighs just over one hundred pounds. She can dead lift two hundred twenty five pounds Witthaya something. She was ready to defend herself so when she saw this intruder she grabbed the closest object tour and that happened to be a small table and he had metal legs. And I'm dragging him Joe Joe and when he's he's down on jumping out all right good for her police thought so too. They posed for a picture with Murphy and posted it to the Rochester. The police twitter account and calling Murphy. Tough as nails. It's the whole world. And everything is awesome Today is Thursday November twenty eighth you are listening to the world and everything in it and we're glad you are good morning. I'm nick and I mary. Reicher next up presidents and Turkeys in eighteen. Seventy three a Rhode Island farmer sent a Turkey Turkey to ulysses s grant at the White House. He continued sending Turkey for the next forty years through ten presidents in nineteen forty seven. The National Turkey Federation picked up that tradition and every year since presidents have welcomed Turkey's to the White House some as dinner others as guests. Here's world radios Paul Butler earlier this week to massive white turkeys.
"cystic fibrosis" Discussed on Skimm This
"It it's Friday November I welcome to skim this we're breaking down racing to get their first then a new medicine just approved by the FDA could be life restraints patients we're here to make your evening smarter let's Super Fast Internet chances are unless you've got a special phone or happen to edgy networks and thanks to years of Government planning Chinese five G. is leaving he actually is why China's been winning this race so far and what that could mean for the US the G stands for generation if you've ever had bad service and drop down to three g faster and five G. is a hot new thing we won't get too technical but and inventing new chips and hardware to receive that data but once that's in place between clicking something on your phone and the cell network receiving that command five G. is needed with five G. Some version of argument has crept up ever since the invention of cellphones Angie are okay we've linked to a few articles on this topic in our show notes okay dreaming crystal-clear episodes of succession on your commute to work it could also revolutionize vision of technology where everything talked to everything else we're talking temperature you get the point by one study there could be five hundred billion Internet of things devices listen which means five G. could turn us all into the jetsons actually in the future I believe it will be very analogy like virtual reality five G. could also help connect really sleek smart glasses well as they race down highways really fast five G. could revolutionize healthcare to this of whether China or the US takes the lead and five g. then why because of the nationwide rollout afford g that gave tech and software companies a lot the lead in autonomous vehicles or so-called smart factories or virtual reality gee lead business leaders politicians worry that five G. networks being built by Chinese a Chinese built five G. Network could make.
FDA approves 'breakthrough' drug for cystic fibrosis
"A breakthrough in a drug to help treat cystic fibrosis the federal drug administration approved a new treatment for the disease caused by a defect in a certain protein that causes a build up in mucus that affects the body it increases the risk for infections and leads to diabetes the drug is called try capped a and is approved for patients twelve years and older A. B. C.'s Michelle Franzen has more for people diagnosed with cystic fibrosis the rare disease has no cure just treatment now a new FDA approved drug called try CAFTA is bringing new promise ABC's chief medical correspondent Dr Jennifer Ashton says a target the defective protein that affects the body but there are some risks clinical trial showed improvement in lung function of about ten to thirteen percent some side effects increase in liver function flu and rash and the price tag for this drug three hundred and eleven thousand dollars a year it remains to be seen how much insurance and Medicaid will cover the drug's been approved for patients twelve years of age and older Michelle Franzen
FDA approves 'breakthrough' drug for cystic fibrosis
"New Hope for cystic fibrosis severs the FDA approving a new breakthrough therapy the drug can treat patients with the most common form of the disease which involves a gene mutation that causes a build up of mucous throughout the body ABC chief medical correspondent Dr Jennifer Ashton it's called try CAFTA it targets that defective protein and thought to be a possible option for ninety percent of people suffering with cystic fibrosis who were previously out of options it is approved for patients twelve years of age and older there's no cure for the disease which is rare but potentially life
Vertex prices cystic fibrosis combo treatment at $311,000-per-year
"Right hi let's take a look at New England business this morning this is been a long time coming yeah it's way ahead of schedule at the same time Boston drugmaker vertex pharmaceuticals gets an FDA okay to begin selling a treatment that will impact nearly all patients of cystic fibrosis this approval for the three drug cocktail called trade caffeic comes months earlier than expected the treatment attacks a genetic error in patients leading to the rare lung disease vertex of course is a worldwide leader in the treatment of cystic fibrosis it's already got three other drugs on the market making billions but treating just a minor percentage of patients this drug is also expected to become a so called
Trump administration seeks to deport children with life-threatening illnesses
"Also want to update tonight on a story that we covered pretty intensively intensively last week. This was a story i broken by w._v._u. Our public radio station in boston and by commonwealth magazine in massachusetts was also picked up thereafter by the boston globe and the miami herald then ultimately by the new york times but when this story first broke it massachusetts it was based on a couple of cases that centered around boston children's hospital boston children's hospital really elite children's hospital and it was almost as an unbelievable evil bit of news when this first started to emerge in the northeast last week there had been no announcement about this from the trump administration the only way anybody learned this trump administration plan was unfolding was when individual families started receiving letters from the trump administration that they had no warning of they couldn't believe them when they received them. They didn't know what to make of them. They started to turn to doctors and lawyers and advocacy groups to help for help with understanding what this could mean but the longest shortest the cartoonish lee evil caricatured bottom line of it as we the merged last week and as we reported here on the shell last week is that the trump administration had had somehow figured out a way to zero in on kids with life threatening illnesses kids with cancer kids with cystic fibrosis kids with rare diseases for which they they need specialized delicate advanced medical care of
"cystic fibrosis" Discussed on This Podcast Will Kill You
"Good stories okay so there is so much research that is going into actual treatments and for a longtime longtime all we could do to treat cystic. Fibrosis was treat the symptoms so if you got recurrent respiratory infections you would treat the infection. If you were having having pancreatic insufficiency then you could give them. Maybe pancreatic enzymes okay. It's not going to fix your pancreas but at least you can digest your food but now oh there's all of these new drugs being developed and tested to target the cause of the disease to target the messed up protein itself so that we can fix this disorder from the start rather than just treating the symptoms wow the biggest difficulty is is that because there are so many different mutations there hasn't yet been a single drug or a single intervention that can work for all of the different types of cystic dick fibrosis if that makes sense yeah so has there been any that work for at least one. Oh there's been multiple. Oh good and i do want to say that i am not assisting fibrosis researcher or expert and so i know that there's so much going on that i know i haven't covered it all <hes> <hes> and so for that especially if you research this i apologize if i don't mention your current research but i wanna talk about some of the things that have had the biggest impacts and some of what i think is the coolest and i'm biased because my friend actually did some of this research which is very cool. That's really week four research okay so one new drug that has been developed and works really great for some people and doesn't work at all for others is called. If a catheter have you heard of it <hes> nova catheter. It's such a weird name for drug. This drug works for people with a mutation not the most common mutation but a a mutation that affects about four people living with cystic fibrosis that one of those class three or four mutations that affects the way. The protein works mechanism of the protein so you have the protein. It's not miss formed. It makes it all the way to the surface but it's not not working properly okay. It's called a gating mutation so advocate after can essentially improve the movement of electrolytes lights across this protein it targets this bad getting protein directly and it's really really effective at essentially just allows for the movement of electrolytes how i don't know the details of it erin. That's just i don't understand how so how if these that's when we get too deep into pharmacology that i can't handle. I'm unsatisfied well. You're going to be more unsatisfied okay but it works. The point is that it works if you have very protein but it's just not functioning correctly. It's not getting correctly if a catheter essentially can bind to that protein in a certain way that allows for proteins to allows for ions to move properly across that protein but obviously that's not going to be effective active for people who maybe don't make any cystic fibrosis protein right yeah okay so there's some other options there's two other drugs i think the pace it and luma catheter tease after <unk> and luma after these both are beneficial for the most common mutation of cystic fibrosis..
"cystic fibrosis" Discussed on This Podcast Will Kill You
"Where you can find his latest album titled no god's only monsters so go check it out and you can find him tweeting at all hallows evil okay so the last eighty years of cystic fibrosis have been big via since its first description eighty years ago. Cystic fibrosis has gone from a disease as of relative obscurity to one of the most research genetic diseases out there. The expected lifespan has gone from six months to over thirty years and and so much progress has been made in treatments and potential cures so i'm hoping errand that you'll tell me some good things about cystic fibrosis and gene therapies therapies and other great things on the horizon. I can't wait to we'll take one more short break <music> <music> so overall. It's estimated that the incidents of cystic fibrosis is about one in three thousand among people of northern european descent. It's very high incidence yeah especially usually for an autosomal recessive disorder..
"cystic fibrosis" Discussed on This Podcast Will Kill You
"To talk about about disease or wellness with our limited vocabulary and i don't just mean you in my limited vocabulary. But how do we know like when we say words like this hurts well. How much does it hurt a lot. Okay yeah we can use more descriptive language. We can use a scale from one to ten but like how do you how can you understand a scale. If you don't know what someone's baseline is one person who's like oh. I'm feeling cruddy. Another person might be like oh my god i'm in agony. I'm dying right now extremely painful. I feel like our own baselines. Change over time a hangover at thirty two is a lot different than a hey over. It was twenty two percent variance and there's such a thing as a hangover at twenty two. I don't i don't think so and i think part of this issue with communicating effectively how we feel or what we're feeling is not just with this limited language but also it has to do with the difficulty in relating to someone what it's like to be you to have your experiences in your memories and the way you see the world because that form so much of how we perceive our own selves and it also how we interpret other people's feelings or words words and that's something that i came across many times in some of these memoirs that i read for the episode that for people born with cystic fibrosis basis. They have not known a life without it so they so you. You can't ask them. Hey what's it like to have cystic fibrosis because it's like well. It's this is what i know. This is how i have lived and and also you like it would be like if they asked you. What's it like to not have cystic fibrosis. It would be the same sort of thing. This is what i know you know and so for that reason we can't. I don't know what i'm trying to say exactly so we i think it's it's very difficult to ever or impossible to ever truly understand what someone else is going through or what their experiences are but i think the most important thing is that we need to try cry. We should try because it builds empathy. Another thing that kept popping up in these memoirs was that other people use the cystic fibrosis as an identifier for those people. It's one of the same their identity is cystic fibrosis and that's not what it is is. That's not the case. That's not what it should be and j whom you heard from in the firsthand sheds a little bit were light on what this is like for him and more about who he is so. Let's hear what j. has to say in his own words. I growing up never enjoyed the tone of cystic fibrosis stuff like i never felt like there is something for me and i wondered wondered if there were other people like me out there. I figured there had to be in this writing..
"cystic fibrosis" Discussed on This Podcast Will Kill You
"You might have guessed but how do we know this okay well. We know this for a couple of different. Reasons one is that it has left traces in old european folklore so there's this old commonly quoted prophecy of quote woe to the child who tastes salty from a kiss on the brow for he is cursed and soon must die no way yes. Are you serious yeah so that's like an old prophecy see that's been found in several different. <hes> old boot's several different things like an old <hes> swiss german dictionary it was in an old swiss almanac mc of children's songs and games i so interesting yeah and we also know that cystic fibrosis is old because there is is this description of an autopsy of a quote bewitched eleven year old girl done in a fifteen ninety five and her pancreas was described up to be swollen hardened gleaming white woo so that's a pretty telltale sign of cystic fibrosis as well okay so those are traces braces. Those are written traces right the real smoking gun of cystic fibrosis ancient or i don't know there are lots of clear alive. It lies in our jeans. Okay so as you mentioned. This condition is caused by having a mutation on the c._f. T._r. arching and tracing the geographic patterns of that mutation and variations in that mutation or the types of mutations can tell us a lot about where and when the mutation probably first appeared. I love it so yes. It was an interesting opportunity to dive into some of the genome like evolutionary genetics vettix which i don't not in my wheelhouse whatsoever very fun. Yes very interesting. Bear with me here. We go for a long time. Despite eight the widespread prevalence of this mutation researchers had a really tough time pin down exactly where it began and how it spread so if you look at research from the early two thousands they're like okay so the mutation probably originated anywhere between three thousand years ago to fifty two thousand years ago ago so it's a pretty big frames yeah and the geographic origin was even trickier to nail down the ancient dna analysis of skeletons skeletons from his earliest seven hundred b c. E did find the presence of the mutation in some samples which is amazing. It's i can't yeah very fascinating fascinating but that still left so many questions unanswered until last year i found a recent study published in two thousand eighteen that claims to have resolved some of these long standing controversies erupt about the origin of cystic fibrosis who okay so these what these is researchers did is that they took d._n._a. Samples from people of european ancestry with cystic fibrosis and they tried to get a wide geographic range of people spanning spanning from all over europe then they could compare their d._n._a. Sequences to see when the mutations likely emerged overlay that with geographic ethic information that they had collected and basically they could make this geographic time line of the origin and spread of the cfpb mutation and just one of the mutations mutations or multiples so this is just the most common okay so this is. Let me find out what the number is delta at five. Oh eight the one yeah so yeah so this is the one that's that's the most the most prevalent in the population so it turns out that after they did this they're most likely scenario. Areo is that the mutation this dutta f five eight first emerged around twenty seven hundred b._c. E which is apparently the bronze age. Okay haven't really you learned what that is yet to age of bronze..
"cystic fibrosis" Discussed on This Podcast Will Kill You
"With new gifts. This can end up leading to things like gallstones or stenosis. It can lead to psoriasis which is liver the failure essentially in people with uteruses and ovaries. You can end up getting delayed monarch which is your first period and in people with testicles pickles. It's really common actually have an absence of the vast difference and that's the duct that normally carry sperm away from the testes so that means infertility ability so that's a lot and honestly that's not even all of it because while this protein is most highly expressed in those type type of glandular tissues it's expressed in a lot of other tissues as well and so- cystic fibrosis can end up affecting your bones which can increase the risk of fractures it can increase your risk for anemia kidney stones chronic kidney disease the list kind of goes on it's pretty serious and it kind of it's a whole body eh situation <hes> so because this is a genetic disorder. The onset of symptoms is very very early. So what would that typically look like in an infant. I assume i just love when you you ask questions that are the thing i want to answer next did not rehearse this is not okay so the next thing i want to talk about is how we diagnose how we recognize cystic fibrosis okay nap excellent so with cystic fibrosis overall you have thickened secretions chretien's and a bunch of your organs lung pinker sliver blah blah blah one of the ways that we actually can diagnose it is that you also end up with increased amount of salt bolt in your sweat right okay so right so that's one of the ways that we can actually diagnose cystic fibrosis grosses and nowadays in the united states in much of europe australia canada. We actually do a newborn screen to test for cystic cystic fibrosis because it is such a serious disease such a serious disorder. We test for it pretty much. Every single newborn warn that is born in a hospital gets a little heel prick and we can test for cystic fibrosis gene mutations. You also could do it by testing sweat sweat conducting. Essentially you re test to see how salty they're sweat. Is which i think is just so interesting and cool that we can do that well. It's it's ingenious. Yeah yeah and what's really great is that if you detect it in a newborn then you don't have to wait until these symptoms manifest best to be able to start potentially treatment or at least preventative measures things like that right but if cystic fibrosis is not diagnosed by by the newborn he'll stick then it's often diagnosed in childhood either because someone keeps coming down with recurrent respiratory infections or we're just has chronic respiratory symptoms so we're talking chronic cough signs of obstructive disease that we can see when we do xrays so their lungs will look look like they're obstructed. When we look at an x ray or you can do pulmonary function tests but on a baby. That's pretty difficult because you have to be like now. Inhale and exhale exhale babies don't know those words and is it. Would it also be seen in like a nutritional like. Would it be obvious in terms of malnutrition yeah so on if you on top of the respiratory and sinus symptoms you can also sometimes get very commonly or used to be more common in very young infants something called mccoy neom ilias which is obstruction of the bowels by mucus plug or it can manifest at first with the pancreatic disease which is basically what you said where you have malnutrition and mal absorption and then the child would present with what they call failure to thrive they are not growing properly etc because they're not able to absorb the nutrients that read and so is this something where again if if you're in a place place where it is not standard to do the heel prick test that again the the type of mutation you have my influence when those symptoms emerge absolutely because it's also very possible that someone isn't diagnosed until adulthood especially if they have a mutation that doesn't result in a complete complete lack of the protein but is just one of these dysregulates proteins or a lower amount of a relatively normal protein so oh in those people in adults who are diagnosed with cystic fibrosis..
"cystic fibrosis" Discussed on This Podcast Will Kill You
"Intestines as well and it's the same thing if you end up secreting really thick nick mucus instead of nice clean watery mucus you can blocked ducks in the same way that you block the airways in your lungs but it in your guts in your intestine. That's going to impaired the silia that are also there from absorbing a lot of nutrients so you can actually end up getting malnutrition and things like that <hes> on top of that it can lead to things like gastro esophageal reflux <music> acid reflux essentially and impaired bowel transit so things moving along your gut the way that they're supposed to because ducks are blocked kind of the hallway hollway along so in really small babies especially this can end up leading to intestinal obstruction on top of the mal absorption that you might be having okay so that's in your lungs and then in your guts and then we have your pancreas which for those who might might not remember is very important organ that secretes a whole bunch of enzymes that are important in digestion and it's a very glandular oregon so that me- degree landy. It's very windy yeah. It's made of a whole bunch of glands. What's an example of a non organ. Your heart muscle your heart. Okay not doing a lot of secreting reading. No no okay okay so if you can't secrete these pro the enzymes that normally do digestion then you're not going to be able to digest your food properly essentially and so that's exactly what happens in cystic fibrosis instead of being able to properly secrete these enzymes your pancreas risk is decreasing thick gunky stuff because the electrolytes and water are not balanced correctly and this means that not only can and you not properly digest foods you end up not being able to absorb really important things like fat soluble vitamins because the pink radic enzymes are really really important in fact digestion especially and fat digestion is important in being able to absorb fat soluble vitamins right so it's not just that your intestines aren't able to absorb. It's also that the pancreas is not even able to help you break down what you need to in the first place exactly right and you might also remember that your pancreas secretes other important things like insulin yeah so if the ducks in your pancreas get plugged up an aren't able to secrete insulin then then you can end up getting diabetes and that's actually a really important aspect of cystic fibrosis that i feel like is maybe sometimes overlooked at least just in common parlance. I think most most people think about the lungs when they think about cystic fibrosis but the development of diabetes is a really serious complication as well diabetes basically is just not enough insulin in your body and if you don't have enough insulin then you can't properly regulate glucose or sugar so then you could end up having really really high blood sugars first and then that can kill you okay so in cystic fibrosis what diabetes looks like is a lack of insulin because you're not able to secrete it so so there's a number of different ways that you can get diabetes. <hes> similar things blocking ducks etc can happen in your liver getting plugged.
"cystic fibrosis" Discussed on This Podcast Will Kill You
"You just click on march now and the other thing is that i i saw this on read. It and i just wanted to share this because i was. I was lurking briefly. Even though i haven't been on very much so on the t. w. kyi sub read it they were there was a recent post or post a while back about what what people wanted to call fans themselves what they wanted to call themselves and there were a male. Let me let me pull up some of these things. Actually one of the top ones was filthy animals of course her affect another one vectors ooh amazing erin de miala jests. Oh my god air infected ooh. I like that i love that <hes> extrema files podcast ask phages quarantine scenes respiratory droplets. I loved that. I wanted t shirt. That says that the word that the herd is also amazing so anyway. I just wanted to tell you that amazing yeah. It was very fun but it's really funny any okay any other business. Actually there is one more thing oh yeah. This is our second to last last episode of this season. Oh my gosh it happened so quickly happened so fast and before you get alarmed by that news. We're only taking a relatively short break. We're coming back on october twenty ninth for the premiere of our season three and so- subscribe to all of our social media subscribe to our podcast so that you see when the new episode drops yes and this is second to last. We're not leaving and you high dry. We've got another excellent episode coming out in two weeks. Yes okay. That's everything now. I think so well then let's get started. Tell me about the biology of cystic fibrosis. I can't wait okay good. We'll take one quick break. <music> <music> <music> cystic favorite said this can be a fun one because we haven't done a genetic disorder before so we're going to talk a little bit about genetics before we get started on anything and on top of that we get to talk about bio chem which just for some reason is one of my favorite things to do on this podcast. I guess so yeah. It's a one of my least favorite subjects. Okay cystic. Fibrosis is an autosomal recessive genetic disorder and it can be caused by. I actually a number of different mutations in a single gene so it's always the same gene that gets messed up somehow but there's a lot of different ways in which the gene can be mutated that end up resulting in slightly different presentations of this disease is or disorder so first. Let's define the words. Autism will recessive because some people might have never heard that that basically means that you have to have have two copies of this gene that are mutated in some way in order to actually have symptoms of this disease so if you have just one mutation your what's called a carrier but you pretty much won't have any symptoms or be sick or have cystic fibrosis. You have to have two copies of the gene gene and so that's what autosomal recessive means in this case cool cool. An autosomal just means that it's not in the x or y chromosome. It's in any of the other the chromosomes right okay so the gene that is mutated in cystic. Fibrosis is called the c. f. T. are very creative. Cystic nick fibrosis trans membrane conducted regulator gene okay. It's the cystic fibrosis gene right. This is a gene that codes for the proteins that form channels through which ions pass so this is where we're gonna go a little bit biochem..
"cystic fibrosis" Discussed on This Podcast Will Kill You
"I think something i learned in old swamp. Thing comic is there's. There's some some guy can see the future so that actually don't remember the context of it but i don't think you're supposed to know the way you're gonna die and like that was a very comic bookie saying like oh. I can't tell you how you're gonna die. That will change your whole life but that made me think for a long time because for years i was sure i knew how it was going to die and it's become blurrier now lake. There's a give it like an eighty percent chance that c._f. Kills me. There's a chance that something else could really come in and take the victory from it but it made me incredibly morbid for so long because if i did if i do have a gift or or superpower superpower mine is <hes> for most people comedy is tragedy plus time. I require very little time. I think most things are very fight when they happened to me. Obviously obviously i think most things are very funny right immediately and i think i made my family really uncomfortable with that. My grandmother who was wonderful to me for so many years was also one of those grandmothers. No you're gonna be fine. Everything's gonna be fine like you. Don't know that anything could happen tomorrow and she'd be like no. You're gonna so much longer than me like you. You don't know that she was right but for me c._f. Is a thing that i know. This is the thing that i do. It requires little to no bravery my my part. I just have to keep waking up in doing that. I have no other choice but i think some people don't like to be confronted with the idea that lake their body could go into total rebellion at any point is mind. Constantly really is it's. It's shocking for that. I have a job and again. It's not easy and there's no shame in not having that if you're dealing with c._f. But it's it's shocking for them to see someone. You're doing quote unquote normal stuff while again body total rebellious at any given point and i just think most people don't like to grapple that when it's literally the only thing i want to grapple with is how i'm going to die when it's going to happen what's wrong with me. <hes> no no no no no no <music> <music> and so you just heard from jay durrani who we had the most fun talking to this week the most fun fund he is an amazing author musician and just all around hilarious person and <hes> we have more more of his interview later in the episode so do keep your ears out for that one yeah it was really thrilling to get to talk to him and we can't wait for you to hear here even more of his story and there's one more thing that you should keep your ears out four at the end of the episode and that's a special song written by j specifically typically for this episode. It's called complete semantic rebellion and will provide the link for download in our show notes. I think it seriously might be the coolest list thing to happen on this podcast anyway. I'm erin welsh and i'm aaron almond updike and this is this podcast will kill you and today we're talking about cystic fibrosis. That's right yes. This is our first genetic one. Is that right sure. I'm pretty sure that it is yeah yeah okay. So what are we drinking. This week are according t this week is the dorothy h anderson yes thus named because there was an amazing researcher named dorothy. H anderson who described ebbed. I think was one of the first people to describe cystic fibrosis and did tremendous amount of research in her life on the condition. What is in the dorothy h anderson well. There is pomegranate soda lime juice a splash of ginger ale to keila always good and you've gotta the have it with assaulted rim and we'll talk about why throughout this episode perfect perfect we'll post the recipe for our core intini as well as the placebo rita which is our non alcoholic version on our website and all of our social media channels and let's see we do have a couple of bits of business do <music> game so one thing that i wanted to mention is that hey we have merch..
"cystic fibrosis" Discussed on This Podcast Will Kill You
"Talk about your physical feelings right and also in terms of of impact on family am divorce rate. These sorts of things like how it's it's such a multifaceted thing where i feel like it's we if we follow this formula every episode right we talk about the biology the history in epidemiology and i feel like that there's so so much more to every single disease that we talk about that we have talked about and in this case it was definitely tip of the iceberg westberg in terms of feeling completely. Ill equipped to tell any version of a story of cystic fibrosis and saying like well. There's also this aspect of it. There's this aspect of it which is even more reason to talk about to say. Hey you know share your experiences and so on but but yeah yeah there's there's a lot of really there's a lot of research in impact on aspects of cystic fibrosis that are maybe not immediately absolutely apparent or fall into the categories of medical or write historical exactly yeah yeah. That's where we stand seems like. It's a encouraging but yeah overall encouraging hard still hard end end heavy <hes> sources sources. I wanna a shadow couple of papers so one is rahman's at all from nineteen eighty-nine and that is the identification of the cystic fibrosis gene and this article is where they identified where the mutation is located located on which gene on which chrome and then there's a twenty eighteen paper by farrell at all and so this is where i mentioned estimating the age so the origin of this most common de 508 new tation and then there are three memoirs that i want to give a shout <music> out to one is called alex the life of a child by frank de ford and so this is a memoir written by a father about his daughter learn named alex who had cystic fibrosis. Another book is called my foreign cities by elizabeth scarborough and so this is a book written by a woman whose news husband had cystic fibrosis and of course can't eat can't breathe and other ways cystic. Fibrosis has after me by our very own jay jay dirani. All of these were incredible. I i've really highly recommend each one of these. I think it's it's just really valuable to read about someone's perspective. <hes> someone's experiences yeah i had to really great reviews actually that are super comprehensive about cystic fibrosis biology and covers a lot of their epidemiology as well so we as always will post the links to all of our sources on our website. This podcast will kill you dot com under the episodes tab and there you can find.
"cystic fibrosis" Discussed on This Podcast Will Kill You
"If you were having having pancreatic insufficiency then you could give them. Maybe pancreatic enzymes okay. It's not going to fix your pancreas but at least you can digest your food but now oh there's all of these new drugs being developed and tested to target the cause of the disease to target the messed up protein itself so that we can fix this disorder from the start rather than just treating the symptoms wow the biggest difficulty is is that because there are so many different mutations there hasn't yet been a single drug or a single intervention that can work for all of the different types of cystic dick fibrosis if that makes sense yeah so has there been any that work for at least one. Oh there's been multiple. Oh good and i do want to say that i am not assisting fibrosis researcher or expert and so i know that there's so much going on that i know i haven't covered it all <hes> <hes> and so for that especially if you research this i apologize if i don't mention your current research but i wanna talk about some of the things that have had the biggest impacts and some of what i think is the coolest and i'm biased because my friend actually did some of this research which is very cool. That's really week four research okay so one new drug that has been developed and works really great for some people and doesn't work at all for others is called. If a catheter have you heard of it <hes> nova catheter. It's such a weird name for drug. This drug works for people with a mutation not the most common mutation but a a mutation that affects about four people living with cystic fibrosis that one of those class three or four mutations that affects the way. The protein works mechanism of the protein so you have the protein. It's not miss formed. It makes it all the way to the surface but it's not not working properly okay. It's called a gating mutation so advocate after can essentially improve the movement of electrolytes lights across this protein it targets this bad getting protein directly and it's really really effective at essentially just allows for the movement of electrolytes how i don't know the details of it erin. That's just i don't understand how so how if these that's when we get too deep into pharmacology that i can't handle. I'm unsatisfied well. You're going to be more unsatisfied okay but it works. The point is that it works if you have very protein but it's just not functioning correctly. It's not getting correctly if a catheter essentially can bind to that protein in a certain way that allows for proteins to allows for ions to move properly across that protein but obviously that's not going to be effective active for people who maybe don't make any cystic fibrosis protein right yeah okay so there's some other options there's two other drugs i think the pace it and luma catheter tease after <unk> and luma after these both are beneficial for the most common mutation of cystic fibrosis..
Genetically Modified Viruses Help Save A Patient With A 'Superbug' Infection
"For the first time. Scientists have used genetically modified viruses to treat a patient fighting a life threatening superbug infection NPR health correspondent, rob Stein. Has the story is it bell. Cornell Holdaway was born with a lung disease called cystic fibrosis when she was fifteen a nasty infection started spreading through our body after she got a double lung transplant in London. Nothing could help her not antibody. Nothing. Her mom, JoAnn says the doctors told her there was no hope devastated to be told. You know, we could well be burying all child was just anyone has a child and never expects to have to bury them this this selves. But then is false doctors decided to try something out of the box therapies called Phages their natural enemies of bacteria. So the doctors found Graham hatful, he's an expert on phases at the university of Pittsburgh using genetic approaches with genome engineering were able to assemble this collection of three Phages that we could then combine tile to use the treatment, they know infect the kill efficiently. People have been treated with Phages before with mixed results. But no one had ever tried infusing genetically modified Phages into someone's body. It's kind of a scary thing to go in and administer treatment. Like this full, which we're completely on new ground. We don't know what to expect. Isabelle's doctor started infusing about a billion Phages into her body twice a day and held their breath. There's lots of things to worry about. And so the very first thing was, you know, does something does anything bad happen. But nothing did. In fact, Isabel started to recover she got stronger and stronger and Isabel who's now seventeen is living in almost completely normal life driving lessons. A school making fake city pool. God ning. No. Now, doctors aren't sure exactly how the Phages might have worked and is about is in cured. She still needs to get fade infusions every day. But the infection appears at least to be under control. I think it's amazing. It kind of shows there is completely. No limit. Until they can come up with really her. Mom, agrees them to be able to just have a little fiddle around with these Phages moins blowing valley when you think about it. Stephanie drafty studies Phages at the university of California San Diego. This is actually a historic moment. Fades therapy, seems to be the most promising alternative to anti-biotics that's on the scene. And this is the first time that genetically engineered fade has been used to successfully treat a superbug infection. Anna human being so strategy and hatfill hope this is just the beginning. What can we do for example to extend this to other types of diseases? The most obvious one is closest which is caused by a related bacterium, and that causes a lot of disease, and that's across the world each year and this very prevalent, drug resistant, strains that are very hard to treat. Now, this is just one case. And a lot more research is needed to see how well phases, including genetically engineered Phages really work, and if they're safe, but with superbugs on the rise and biotic losing their power. Researchers hope Phages could help save more
Phages, Graham Hatful And Rob Stein discussed on Fresh Air
"For the first time. Scientists have used genetically modified viruses to treat a patient fighting a life threatening superbug infection NPR health correspondent, rob Stein. Has the story is it bell. Cornell Holdaway was born with a lung disease called cystic fibrosis when she was fifteen a nasty infection started spreading through her body after she got a double lung transplant in London. Nothing could help her not anti-biotics. Nothing. Her mom, JoAnn says the doctors told her there was no hope devastated to be told. You know, we could well be burying all child was just anyone has a child and never expect to have to bury them United the selves. But then as doctors decided to try something out of the box thyroid is called Phages their natural enemies of bacteria. So the doctors found Graham hatful, he's an expert on phases at the university of Pittsburgh using genetic. Approaches with genome engineering were able to assemble this collection of three Phages
Box Office: 'Dumbo' Lands at No. 1 With Soft $45 Million
"I'll look at what was big at the theatre box office is this weekend. From USA radio's Wendy king. A former Disney animated feature is brought back to life. The remake of Dumbo is number one coming in at forty five billion the scary picture directed by Jordan Peele us is number two at thirty three million slipping down the number three captain marvel starring Brian Larsen and Samuel L Jackson is that twenty million coming in at number four the romantic novel about cystic fibrosis five feet apart is at six million. And at number five that true story of what happens to a Planned Parenthood clinic director, the movie unplanned comes in at six point one
'Captain Marvel' soars with stellar 2nd weekend at US box office
"At the box office. So you're not from around here. It's hard to explain. Shining all other movies this weekend at the box office, captain marvel can then at number one starring Breen Varson and Samuel L. Jackson, total sixty nine million dollars. Number two. The new animated feature Winter Park at sixteen million the romantic comedy about a couple of with cystic fibrosis five feet. Apart comes in at number three with thirteen million. How to train your dragon the hidden world drops down to number four at nine million and another sequel from Tyler Perry and the family funeral drops down the number five with eight million
'Five Feet Apart' Review: Ailing Teenagers Live Dangerously for Love
"Now we're talking about movies. Tim's website is celluloiddreams dot net. So Tim what have you seen? I think there's one that reminds me of another movie that came out a few years ago at least in the description, it's called five feet apart. Isn't there something like that that had to do with these teenagers? Oh, the one about. Yeah. Piece distinguishes itself because well, first of all begins and ends with a speech spoken by one of the leads a teenager played by paley Richardson, who has cystic fibrosis, and it's a rather inaugural speech about the importance of touch. You know, how we as humans need it as much as the air, we breathe. So this is a story it's romantic drama about two teenagers who have cystic fibrosis. The other one is played by Cole sprouse. How'd you like to have a name Cole sprouse? I'm sorry. I didn't change that name probably not any way to teenagers with cystic fibrosis. They fall in love. And the tricky thing about people afflicted with this diseases. They can't touch each other for fear of some kind of potentially fatal bacterial infection that can be transferred from one to the other. Anyway, patients with this disease. They have to stay at least six feet away from each other and the title here five feet apart. I think. Is something that the two characters come up with it has a little risk factor involved because of another speech somewhere that one of them gets about a certain amount of or certain element of risk being necessary to look, and I really wanted to like this movie. I went in there expecting to because the distributors CBS films has been responsible for releasing some really good movies over the years. Like last year's it turned east gate and the first half hour is actually quite good. When the impossibly cute characters are introduced in the plot points of what I just talked about worst stylish in the narrative, and you learn about cystic fibrosis by watching this film. So it has that going for it. But the movie about half an hour into it it descends into this truckload of cliches repetitions on this team of I love you so much. I wish I could just touch you and literal hysterics about death and dying in survival guilt. So you know, when it didn't try it was kinda sweet and charming and the actors were all good. But when it tried it became borderline I rolling, and it was probably the least favorite of all the films. Yeah. I know the script was just it lack. I think it's strictly a movie for teenage girls, and maybe a teenage gay boys because our female protagonists would know it has a gay best friend. Fibrosis right is a good line in the film. Pat smoke is spoken twice. Did they say it's just life? It'll be over before you know, it, and that's kind of how I was feeling about the movie. It's just a movie it'll be over before you know, it. All right. Let's skip that one then that's five
What are the potential health benefits of gene editing?
"Last year. A Chinese scientists shocked the world by disclosing hit created the world's first gene, edited babies, and John Ahuja talks to Robin level badge a developmental biologist geneticist about the controversy and about the potential for easy to use gene editing, tools such as crisper Cas nine to revolutionize diagnostics, drug discovery and the treatment of disease. Everybody has become very familiar with the story of the gene edited babies that came out from China last year. So these were apparently, the world's first genetic babies created it really triggered a scandal. Do we know what's happened to the scientist and author the twin baby girls that have been born? We know nothing new particularly drunk you. Hey, all vote voters JK is currently in Shenzhen where here's university is. I think he's in a an apartment owned by the university and the stories in the Chinese press that the gods. What we don't know who those gods are whether the university outs police, whoever they are. We don't know whether he's under house arrest or whether he's free to move around. He actually emailed me and said, he's all. Fine. So the impression is I think he's trying to give me that he's not under arrest. So the goals may be that to protect him because I know he was receiving threats during the become fronts that we had last hole. I can say he's fine. About the welfare of the twin girls. Lulu a Nana be concern. We know nothing about that. Jake himself was very keen to stress that that privacy should be maintained that they should be protected and density, not known on. We'll this likewise their parents. There is an investigation being launched in China by the ministries of sciences on administrative health. They presumably will want to find out whether what he's claimed to have done is actually the case. And so they would need to take DNA samples from the two babies, and the parents, but I hope they do it in a sensitive way. These are just two little babies. They should be cheated just as that and not subject to anything bad at all. They have novel mutations in this, gene L five, which is known in some cases to confer protection, it mutations in that gene can confer protection against HIV. And that was the rationale behind the work. But Jake I did was to try and make these children immune if you like to HIV because having trophy in China of even being a member of a family where one of the family members house HIV as in this case, it's cheap very badly. Families the stigmatized the children wouldn't be allowed to play with other children simply because of that for example. So that also sized about another reason why the density needs to be protected because of that situation too. But we hope is that they will teach it to normal little girls. And that's it. They have mutations in the gene that novel mutations because of the way he did the dean of editing quite poorly. We have no idea what those mutations will do but all of us have mutations. Every new generation that forty two eighteen you mutations in genome. So just treat them like normal children. Keep a watching eye on them. See what happens say watch this space on the genus babies story. But of course, may scientists involved in genome editing, as you prefer to call it on not involved in trying to change the course of human evily Shen by creating these genetic mutations or changes that will then be passed on down the family line, as I understand it the real excitement for genome editing is in the lab working with adult patients, perhaps with single gene disorders also excitement with drug discovery and so on so let's talk about that. Because I know that you were involved as developmental biologist in the aren- decide of things does your work focused on the creek. We have a number protecting many projects, but let me decide about one of them jeans have to pulse to them. They have the part that encodes the protein is going to do its job for it. But it also has a part but controls when and where that gene is active so normal genes, right? Even all cells over time, they all cell type specific, and they can be. Stage of development specific they can be on the active of particular time in the embryo or any adult we've been using the genome editing methods to try and understand bisect light. This regulatory region for some jeans and one in particular, cold sulks nine so soaks nine is gene that has many roles in developing embryo, but one we'd been working on for a long time as its role in sex determination. So whether you become male-female soaks nine is really critical to give rise to the development of testes particular cell type was critical for making tested so mutations in soak nine can lead to sex reversal to give ex wife female development instead of male development. But it's a really complex gene is active in many different cells. And it turns out that it has an enormous regulatory region, which is really complex and we wanted to try and dissect that say you have specific sequences you can refer to enhance or sequences where other proteins transcription factors in Toronto. With those. To tell the gene to be active or silent. And so we had fun using variety of methods number of candidate regimes run home of for soaks nine in the process of making testes we had about thirty two different candidates. We started with and we use number methods to try and reduce that number down. We came down to four but looked like they were very promising, and we use genome editing methods to inactivate each of those in enhances and turn to basically delete each of those regions of DNA intern unfound one of them, which is located very long distance away from the protein, coding Potter, gene itself. Some six hundred fifty thousand base pairs away which is big distance. When we did he should that particular in Holzer it inactivates, the, gene. So we got X Y females. So even though you have this really complex regulatory region. Spread over many millions of base pairs and stuffed full of different regulatory regions. Turns out, but just one was essential for expression in the developing gonads said that they can rise to test. What's the next female? They are crumbs Emily mail voicemails scream saying about they've developed as a female in this case in the embryos. They are indistinguishable from normal females stabbed genotype female genitally. This is women. They have the mice. Not women they develop his females in humans, of course. Yes. You would also have cases where you have extra female development. So they look female, but chromosome Yuda male. They will be infertile because the germs house of the eggs in the next female that do very well, they get lost early on. So they will be infertile. But otherwise, they look and generally behave and all female month thing, I should ask you to do been is to explain very briefly. What's crispy cast Moines editing, as can you tell us how the crisper cast nine genome editing system works, very briefly, please, okay. It relies on making breaks in the DNA usually a double strand break in the day, and to do that you have an enzyme called cows nine, but you have to have a way of getting not enzyme to the right place in the genome. And that's the guide are a component. So the RA you design it part of the irony is designed to be complementary. So to match up with the DNA sequence that you want to cut the other part of the RA guide are on a has a sequence of interacts with the cows nine enzyme protein. So when you have both together, and you crisper is not linking crisper yours. Crisper is a shorthand term for the guide our nights for the irony. The name crisper comes from origin. Bacteria is not really relevant to. Use to the guy Rene takes the Cas nine enzyme to the right part of the genome. The enzyme an cups, the DNA and processes that occur in also to repair broken deny then jump in and try and repair it now in the simplest form of technology. You just making a break in the DNA actually, Germany making a mutation in a gene, you have a process called known homogeneous end joining horrible term, but it basically just tries to stick the broken ends DNA bound together again, but often you have a little mistake made and that can be sufficient to an activator, gene. This is incredibly useful in the lab for studying gene functions say rapid and easy. Now, if you want to make them more precise alteration in the genome sequence DNA sequence on that can be anything from a single letter up too, many many thousands of letters of code then you have to use a different DNA repair mechanism cold Hamasi directed repair. This requires a third. Component to be introduced to the time time, which we confined to his DNA template. So this has to pulse. Each end. You have a bit of DNA, but complementaries of that's the same as the gene in which you want to talk it. And then in the middle. You have a sequence that you want to replace the one that's in our ready with and if you have those three components together. Now, the cows names aren't the guide our day on the DNA template the direction repair mechanism will not work, and it will substitute. What was in old template into the gene it is quite an incredible technology in it? There's a third method which I wanted to tell you about which is really exciting, which is cold base editing. Now, this allows you to change one base pair one letter if you like in the code, but it works without making a double strand, cutting the DNA, it changes chemically if you like one letter to another then it's partner also then has to change. Change. Otherwise, you have a little loop in the DNA, and that relies on a different type of DNA repair maximum to do a little bit. But given that about half of single, gene disorders or calls. In fact, by single base pair changes mutations in the DNA, this new methods looks really promising because it doesn't come with the baggage that some of the other methods have of creating unwanted mutations. So you get just what you want. So it could well be about safer method at least when you're dealing with single based mutation. Tell me how genome editing is being used in the lab, and perhaps with patients, it's really exploded. In basic research. The use of genome entertain particular the crisp cows nine methodology, it's very easy to use. That's one reason relatively inexpensive to use. But it's just so powerful and it's a rapid. So we've had ways of altering genes in cells in coach. Uh-huh. Or in model animals that we use for research like mice methods to alter genes in many years for decades, but they will always very inefficient very slow to make a mouse carrying specific mutation in a particular, gene, it really used to take well over a year to do that we cannot do it in the motto of a couple of months. So it rapidly speeds up the way that we can do research to study the role of specific genes or pulse of genes during development in my case or for physiology over brain function, or in cancer, it's really speeded up things enormously and made it cheaper in terms of things like the possibility of using genome editing to treat patients who already have a genetic disease or council something like this again, that's really looking incredibly exciting. We've had some somebody, gene therapy. Also for decades, just explain what semitic therapies somatic cells are. Basically any sewn in the body apart from the jumps health germ cells being themselves who are going to give rise to sperm or eggs that would allow any genetic changed repulsed on to subsequent generations. So a typical somebody will be a skin owl muscle tell or bone cell brain zone. So there are whole ranger Nettie disease is that can compromise. The ability of particular cells to function or the politics function things that the blood'll sickle cell disease will be catalyse Mia, you have disease like cystic fibrosis or muscular dystrophy affect lungs muscles opticians, in many cases, these G two single mutations Chinga, gene. It's affected by small mutation affecting that, gene. So the genome editing methods can be used to correct, gene defects, or in some cases, find ways around of getting another gene to become active to replace the gene that's not working. There are now probably around twenty or so clinical trials that had been launched. Using the female amenity methods to try and treat individuals who have think disease of this soaks. I'm single, gene defects.
Serena Williams Sports Gatorade Dehydration Patch
"Business wars daily is brought to you by Pitney Bowes, and send pro online shipping can be complex with the uncertainty over costs and deciding which carrier to use plus tracking your packages things can get confusing. Stay tuned to the end of the show to find out how Pitney Bowes and send pro online can save you time and money and to get a special offer just for listeners of the show. From wondering, I'm David Brown. And this is business wars daily on this Friday January eleventh. Serena Williams is analyzing her sweat while playing tennis in the latest ad from Gatorade. The tennis star sports. A patch that passes sweat through food coloring giving an instant easy to read picture of hydration or lack thereof when colors on the patch change. They signal levels of dehydration sort of like a mood ring for electrolyte loss. Williams told fast company, she was excited by the promise of the new wearable technology to extend athletes careers, the patch invented at Northwestern University and developed with Gatorade sports Science Institute has been tested by professional, athletes and college swim teams. It's also being used to test babies for cystic fibrosis. The patch is the latest volume Gatorade attempt to rebrand itself. From a sports drink to a spa. Ports fuel from something. You might want to something you have to have Gatorade holds about three quarters of the eight billion dollars sports drank market. So why go to the trouble inexpensive developing sophisticated wearable technology. Well, Gatorade needs some fuel of its own. The brand sales have been slowing lately as consumers turn away from sugary drinks in favor of more natural beverages. That trend has also hurt. Gatorade rival Coca-Cola which owns PowerAde last year. Trying to solve that problem. Coke took a stake in body armor, the up and coming sports drinks startup is backed by basketball star Kobe Bryant body armor uses coconut water, which the company argues is not only more natural than its chemical heavy competitors. But also lower in calories the brands market shares tiny, but it's been growing faster than either Gatorade or power aid for Coca-Cola investing in body. Armor represented a move into the future for Gatorade. It's packed system is also seizing on the opportunity to evolve Gatorade says a retail version of Serena's sweat patch costing three to five dollars will be on the market soon. And that may signal even more about Gatorade future as a brand marketing health technology that just happens to come with sweet drinks, the think about that. The next time. You're practicing your serve. From one to read this business wars daily pay fascinated by the long running battle between coke and Pepsi. Check out the whole story in our six part series on business wars, this week's episodes were written edited and produced by lane Appleton grant, Ginny lower is our editor and producer our executive producer is Marshall Louis reated by or non repes- for one. I'm David Brown. We'll see next. Shipping can be complex with the uncertainty over costs and deciding which carrier to use plus tracking your packages things can get confusing. Now, there's a better way to ship central online by Pitney Bowes with simple online. You can easily compare USPS and other shipping options in an all in one online tool, you can print shipping, labels and stamps on your own printer and track all of your shipments as well. Plus when the US postal rates increase on January twenty seventh you'll still be able to access savings of up to forty percent off USPS priority, mail shipping and five cents off. Every letter you send just by using central online. Simple online is only fourteen dollars ninety nine a month, and you can get a free thirty day trial. When you visit PBA dot com slash b w daily. You'll also receive a free ten pound scale to help. You weigh your packages and accurately calculate the cost of shipping. That's PB dot com slash. WD early and big thanks to Pitney Bowes in central online for sponsoring this show.
Inhaled RNA Might Help Heal CF
"This is science Americans sixty seconds science. I'm Christopher Dodd Yata. Genome is sorta like a library with each gene and instruction manual for making proteins, the bad news is you can't return a book and take out a new edition. But the technique of gene therapy allows you to revise your copy of the book, so to speak giving you the ability potentially to make new in different proteins now scientists have experimented with a new way to make text revisions by inhaling the changes they tested the concept by having mice breathe in genetic material called messenger. Aren a or m Aren a for the test. The Amarna included instructions to manufacture Luciferian the stuff that lights up in firefly's. The researchers packaged the Marna with a degradable polymer to trick the mices lung cells into accepting the package, and once the cells gobbled it up they began to glow proving that inhaling 'em are in as an effective way to kick. Start the production of new. New novel proteins like the firefly enzyme. The scientists also repeated the experiment in mice who sells had been genetically engineered to turn permanently read if they received a copy of MR in a making it possible to count the proportion of cells affected by a dose. The proof of concept is in the journal advanced materials, of course, the point of all this isn't to make mice glow. Instead one idea is to use this technique to help cystic fibrosis patients because people with CF have genetic mutation that causes a build up of sticky mucus in their lungs. Several of the study authors work with publicly traded biotech company called translate bio and that companies conducting phase one in two trials to determine if inhaling messenger are in a could provide a genetic fix for cystic fibrosis, regardless of the particular case of CF, the Luciferian example shows that inhalation genetic therapy could be an inspiration. Thanks for listening for scientific American sixty seconds science. I'm Christopher Don. Yada.
"cystic fibrosis" Discussed on Newsradio 700 WLW
"Weeks to get the puppies. Think of all of the effort. He is putting into getting two dogs. More Ohio, driver's license is more. You can get a day. I have so and I've got a driver's license. I haven't got it renewed recently bunions dance a pain in the neck to get a dog is a big deal now bidding dooby declared mentally ill and not to be a big deal. Now, that'd be like, okay. Before we take away all your rights under the constitution. We've states better able to prove that you're mentally ill suffering from diseases effective such a character and impairs your judgement about about about other those people's rights. You get according to John cranley liberal out. You get rid of them. They're crazy. They don't matter. No matter what if you got cystic fibrosis like you said, an Argo, mental illness, isn't something you choose. It's some it's a biological chemical misstep, you know, and it can be corrected with medication. With medicare? But if you if you start marginalizing people and their rights based off something that they are born with then couldn't you say the same like, well, he has cystic fibrosis. He's not allowed to own a gun got a couple of family members mental difficulties and they're on their meds. Pretty good. Everybody has had family member. Because it's so common. Even though this country doesn't do a really good job of handling mental illness having resources for people and their families to get them help. Unbelievable segment gave me some sports and make it fast..
Multi-gene test may find risk of heart disease and more
"Researchers have developed a, new way to analyze genetic data AP's Jackie Quinn reports it may one day help, identify people at high risk of combinations like heart disease and diabetes genetic testing right now mostly focuses on rare mutations like ones. That can cause breast, cancer sickle cell disease and cystic fibrosis now research With
Vertex gets approval to treat toddlers with cystic fibrosis drug
"For the first time. Ever toddlers will be able to take a medication for the treatment of the most common form of cystic, fibrosis the FDA approves. The drug from. Boston's vertex pharmaceuticals to, treat, kids between the ages. Of two and five they drug is already on the market for older children the. Approval based on the results of a pivotal, clinical trial of the drug or Camby
Newly-discovered type of lung cell has central role in cystic fibrosis
"There's still a math problem. Okay. Still a math problem. You still need donor, you will, you know, or an organ donor situation and to to do the providing. So. Well, we're getting better. I mean, we're getting closer if still very far away. I feel like we're getting closer to to being able to three d print, organ, scaffolding, right? We're, we're getting incrementally closer which means if we can get better at this side of it, then. You'll need substantially less lungs. So we're perfecting the system here and then incrementally close to a lot of things in this world. But, but yeah, the. With scaffolding thing is, is is a real thing that's going to happen in question is, do you need indeed? Do you need the scaffolding? Do you need it to come from a human pigs? Have the reason it use pigs as test animals is because they have very similar physiological and anatomical parameters. So we use pig hearts for pig transplants. Is it possible that we could also use the pig lung scaffolds that have been seated with the human, the human lung cells? Right. So would that be in inbetween until we can three d. print the scaffolds? And I think a big part of it is the fact that it came from an unrelated animal because that that does that does indicate that you won't have the typical organ rejection at problems, which yeah, I think you're right. I think it may not matter then. Protein scaffolding doesn't appear to have any of create any that means system reaction because it's loaded with the tissues from the donor. So it doesn't matter. Maybe we're already there. Maybe this is maybe this is it. Maybe we don't have to wait for the three d. architecture. Maybe we can use pick lungs. Yeah, at least it's a good stopgap potentially if it works in humans and then the next the next question is, can we use this methodology? I mean, the big issue here they're talking about lung damage and has being a leading driver of the need for these lung transplants. And the fact there is an organ shortage. Well, if your lung has something like cystic fibrosis, which is the result of a genetic issue, if you don't want your new lung to be seated with your cells, even though it's tissue matched because your they just this week, published a new paper about a new, a new cell type in the lung that was found that they think is one of the main causes of cystic fibrosis, which might be responsible for it. So the question is then, can we find that cell type genetic and you had an or genetic engineering genetic, you know, change fix the mutations or I mean, what can you. Do you know? How could we have my floor, right? Yeah, you might be able to you. You can. I mean, it's not might. This is beyond Mike. You can do that. Yeah, you can absolutely do that. We are doing it. Oh, it's so exciting is so exciting. This is this is pretty exciting news. I had loved. I mean, the fact that they had up to two months revival of an animal. Then they got this is that's how long they even human assumed that they would be able to look out, but are they could have gone much much longer or you know they were, they were studying, was the integration adapt serve the healing process in there as well. But the way in that's that after two months, I suppose you're just looking at the, you know, at a lot in and it, it's not really as interesting. I love looking at just a lung. That's exciting. Yes. That's great. Let's see. Moving on forward, what else do we have other than the we're saving the world with bioengineered lungs that's happening. Well, want to talk about some bones. Yeah. Where did vertebrates come from. This is a big question. Where did the bones of vertebrates come from? And researchers have several have had several hypotheses about where the bones of Vert of vertebrates us and our ancestors where they came from. And so we know that there early on four hundred million years ago, the evidence for our skeletons early evidence for the volition of vertebral columns and skeletons were found in fossil fishes called hetero Strachan 's, hetero dragons. I love saying some names of things..
Dr Francis Collins, National Institute Of Health and Nell WW discussed on 24 Hour News
"W w j beat reporter Jeff Gilbert WWE news time is seven twenty two a, cure for cystic fibrosis could be on, the horizon WW Jay's Laura explains Dr Francis Collins who is now the. Director of the National Institute of health worked at the university of, Michigan he was one of a handful of scientists who discovered. The gene that causes cystic fibrosis in one thousand nine hundred eighty nine the, basic cause of cystic. Fibrosis have now led us to the point where there are really effective drug treatments that are emerging for his contribution. To sign Science, he is being awarded the. Twenty eighteen Warren Alpert foundation prize. Is efforts bringing the possibility of a cure much closer I. Think ultimately give another decade or two we, may figure out a way to actually cure the disease by a direct correction of. That misspell gene using something called gene editing gene editing is done by cutting DNA Laura. By Nell WW w, j NewsRadio nine fifty years a new, warning this, morning out about gene editing, the findings involved the safety of the gene editing tool called crisper. Cast nine it's a molecular scissor that can be used to cut DNA but the study published today in the journal nature biotechnology shows. That the tool can wreak.
"cystic fibrosis" Discussed on WDRC
"And press both the dan and myself is that you are working on and this will be a big success in xinjiang berry you are working on a benefit for the cystic fibrosis foundation how come that particular organization well a friend of our family sign has cystic fibrosis and it's always been important to our family to support such a wonderful cause and for me it's so important because i'm a singer i have my lungs and their powerful and their big and the fact of being born with horrible lung issues and infections and end a chance that i wouldn't live that long i'm i'm twenty eight some of these people's lives are so short ed it's such an important caused make sure we can raise money to help these people live longer and it it that's wonderful that's fat and all the proceeds from this event will be donated to the cystic fibrosis foundation is that right that is correct everything at tickets only 20dollar yes so take us through the night what what's going to unfold could it be friday august the 25th right correct bury us seven o'clock yes well it's going to be first off there will be candlelight not real keddell edelbe fake had we're not very real fire at sage i it is an interesting event we're going to have have there will be some opera obviously as a may um there will.
"cystic fibrosis" Discussed on The IVY Podcast
"Cystic fibrosis foundation pretty much knows every family in member states kid with cystic fibrosis i never knew anybody with cystic fibrosis after a high school is they're all dead i haven't woman worked for me ten years ago she was thirty she has said that there are she's like twenty actresses an office i said tell me about your wife says take twenty five he said all agassi i play rugby has the gas copenhagen earlier next day on the phone call system and she's on the call andrew i she says oh by the way is our union i assisted by roses present back in my office i said earlier this morning i just ask you got your wife mention do you realize that nine wounded buddy your age as preparations for mild down because they died at fourteen you're twenty in your life expectancy now does worry is not even all the top of your excu who ns progress because the cystic fibrosis foundation raise money from all those families and invested small biotech they've got bigger vertex and with vertex developed the first drug id twenty years to ensure cystic fibrosis in his segment of the population cystic fibrosis check for their share of the world of that droid they invested nine hundred fifty million a fifteen year what did they get three point three billion pak which is twenty years of donations to assist offenders from partnering iranian patients families who would lead the kid to those charts and that's why the small companies are the answer because patients thumped in their eyes they do with small companies look at the except when i was adviser i said fired you need to take money from the small donations.