18 Burst results for "Severe Combined Immunodeficiency"

"severe combined immunodeficiency" Discussed on NEJM This Week - Audio Summaries

NEJM This Week - Audio Summaries

04:53 min | 4 months ago

"severe combined immunodeficiency" Discussed on NEJM This Week - Audio Summaries

"Cardiovascular events or major bleeding between patients assigned to eighty one milligrams and those assigned to three hundred twenty. Five milligrams of aspirin. Daily colin beijing from the university of oxford united kingdom writes in an editorial that the publication of the results of the adaptable trial represents a major step forward in establishing the pragmatic trial as an investigatory tool in the united states. First and foremost it provides proof of principle that large pragmatic randomized. Trials can indeed be conducted in the united states yet. The ultimate aim of the trial was to distinguish the clinical effects of two aspirin doses. Here unfortunately adaptable was west successful. Not because no differences between the two dose groups were observed but because of the substantial dose switching among patients bias arising from this degree of crossover could have obscured a true difference in efficacy or safety or both and moreover it is also not possible to conclude that the lack of any significant difference between the two dose groups implies equivalent of the effects of the doses the adaptable experience is a reminder of the importance of certain fundamentals when designing and conducting randomized trials compatibility between treatment groups good adherence to assign treatment complete. Ascertainment of outcomes and unbiased statistical analysis are all essential. Adaptable is a major achievement. However because it has shown a method of conducting trials efficiently and at low cost in the united states and the method can now be adapted and used more widely. This should allow many more clinical questions to be answered with obvious benefits to health. Care consumers antibiotic therapy for six or twelve weeks for prosthetic joint infection by li na from the university hospital but i know tool france prosthetic joint infections are associated with considerable morbidity the management of prosthetic joint infection usually consists of a combination of surgery antimicrobial. Therapy the appropriate duration of antimicrobial therapy for this indication remains unclear. This open label randomized non inferiority trial compared six weeks with twelve weeks of antibiotic therapy in four hundred. Ten patients with microbiologically confirmed prosthetic joint infection. That had been managed with an appropriate surgical procedure. Persistent infection occurred in eighteen point one percent of patients in the six week group and nine point four percent of patients in the twelve week group thus non inferiority was not shown non inferiority was also not shown in the per protocol and sensitivity analyses. The investigators found no evidence of between group differences in the percentage of patients with treatment failure due to a new infection probable treatment. Failure or serious adverse events among patients with microbiologically confirmed prosthetic joint infections. That were managed with standard surgical procedures. Antibiotic therapy for six. Weeks was not shown to be non inferior to antibiotic therapy for twelve weeks and resulted in a higher percentage of patients with unfavourable outcomes. Autologous ex vivo lent viral therapy for a denison dam aced deficiency by donald kohn. From the university of california los angeles severe combined immunodeficiency due to dennison dominates deficiency ada skid is a rare and life threatening primary immunodeficiency patients with ada skid have profound lymphocytic pena impaired immune function and failure to thrive and without treatment die from infections by the age of two years. This research project evaluated the safety and efficacy of an investigational anti viral gene. Therapy composed of autologous cd thirty four. He met a poetic stem and progenitor cells. That had been genetically modified ex. Vivo with a self inactivating. Lente viral vector in three studies to in the united states and one in the united kingdom fifty patients in total were treated with the gene therapy overall. Survival was one hundred percent in the studies up to twenty four and thirty six months event. Free survival was ninety seven percent in the us studies and one hundred.

Five milligrams three hundred fifty patients six Ten patients ninety seven percent six weeks donald kohn one hundred percent one hundred twelve weeks six week one percent four hundred twelve week two dose united states two years both First
"severe combined immunodeficiency" Discussed on America Dissected with Abdul El-Sayed

America Dissected with Abdul El-Sayed

05:13 min | 4 months ago

"severe combined immunodeficiency" Discussed on America Dissected with Abdul El-Sayed

"Normally therapy sessions are totally confidential. But dr hillary mcbride opened the doors behind sessions with our long standing clients in other people's problems from cbc podcasts. This is what people sound like when they talk with someone. They trust about healing addiction. Parenting stress on in the family and other topics that feel so timely as we come out of this difficult time you can find other people's problems everywhere you get your podcasts Cdc updated guidelines to allow vaccinated people to congregate without masks both outdoors and indoors. The fda in cdc of also authorized the pfizer vaccine for use in children aged twelve to fifteen doctors and scientists reported a breakthrough in treating children with severe combined immunodeficiency otherwise known as the bubble babies because it forces children to live in a bubble or risk infection. They used the disabled version of the virus. That causes hiv delivered genetically modified immune cells to the child's body. This is america dissected. I'm your host. Dr abdul say. Last week i sat down with representative corey bush to talk about her fight for black lives from the bedside.

Last week abdul both twelve dr hillary mcbride fifteen doctors america corey bush hiv cbc cdc pfizer
"severe combined immunodeficiency" Discussed on Dermalogues

Dermalogues

04:24 min | 1 year ago

"severe combined immunodeficiency" Discussed on Dermalogues

"Disorders like hyper syndrome and severe combined immunodeficiency. Thanks. The first thing that pops into my head is you don't really get and again me being a bit strict with the definition of a neo nate. You don't get a lot of neonates presenting with severe a top of dermatitis that's like when day old or a three day early. It's too early and so by definition. And we do see these babies I'm just thinking of one right now who present that early and with chest terrible refractory ads. Is Start, thinking about it right from the very beginning because the majority of kids will start present. You know two months old six months old eight months old and beyond when they're presenting early in its severe, you do start to wonder about I. Don't know I hyper g duckie whatever and and so. initially, you treat those babies as exe- manage challenging too because of their body surface area you worry about absorption. Topical Corticosteroids etc and Parents Steroid Phobia and when the child is twelve, the issue imagined L. Yes. Yes. Yeah. So So, but I think in general, the previous principles apply. So a good history and physical clinical signs that make you worried about this baby is size So if you have a kid who's on the third percentile or lower and you have to look at the size of the family as well too but it babies not feeding and growing the way we expect them to. certainly a baby that's had antibiotics in that time period you start wondering about you know, are you susceptible to infection because you're genetically susceptible or is it just as your skin barriers is so denuded but babies like that are going to get a workout. and. IMMUNOGLOBULINS and. Possibly, like I will tend to do. A brief immune workout I don't like to take tons of blood from these little kids and want someone who's really well versed in what they're looking to to June appropriate assessment. But initially, I will do things like goblins and just to see hyper I G. E and and then a referral if he kanye onto immunology but I think if the baby to two point, I got a baby's presenting early with severe I. Think You'd think about it right off the bat gay so yet. So that is the clue. The severe early station is the clue not this is the position clues a require. I guess I'm thinking along those lines in this isn't a question. The residents had this question that I have an sure. Some of the people have it too. So what I was thinking about was you know sometimes you do get called to see a baby with respect to to a diaper rash. And so. When you're seeing what makes you think about either just sort of classic baby diaper rash versus maybe a ridicule Oh Earth Serie A history say us versus maybe an accurate dermatitis in Pathak with with there be some clinical clues when you're looking at that because you know sort of Red Scaly inguinal folds. Or Yeah. So how do you? How do you approach that? So the diaper dermatitis that we typically see, we'll be erosive especially if it's Jacquet's sort of diaper dermatitis in its vault and history is important because the parents will be using almost no barrier protection at all and it's sort of progressed. So the and the presence of satellite lesions and that sort of thing helps you sort of put it in the regular even though it's really bad in the regular category, I think Acronym Montana Center Pathak. PGA, is it big for because? It's so sorry as of okay so that perenniel involvement often has this and Aniela sort of. Form well defined book. Without any satellite lesions to speak of, and it often will have some periods of erosion throughout it, but it tends to be much more story as a form..

diaper rash severe combined immunodeficien hyper syndrome Acronym Montana Center Pathak L. Yes Pathak PGA Jacquet kanye G. E
"severe combined immunodeficiency" Discussed on Psychology of Entrepreneurship

Psychology of Entrepreneurship

04:59 min | 1 year ago

"severe combined immunodeficiency" Discussed on Psychology of Entrepreneurship

"All an entrepreneur is someone who starts a business from nothing and leo named the generational grew by the washington post in an interview at cornell. University said my definition of an entrepreneur is someone who is creative is compassionate who is a visionary who sees opportunities and seizes the opportunities and that is what makes the difference and then who sticks with it. It takes a lot of persistence and a lot of resilience. You get yourself knocked up a lot. You hit your head against the wall a lot and you have to have the courage and the vision to go with it not to stick to the rule book. Change the rule book if you have to but if you have a vision and you know that it's going to impact the world and it is going to help people in some way by giving them jobs giving an opportunities a new product and sticking with it. That's what an entrepreneur s. I've had the opportunity to speak with some pretty incredible entrepreneurs in leaders over the past couple of years specifically for the show learning about their journeys and finding out how their brains are wide in the hope that i could understand mine and in turn give you something to ponder about every week as i get exposed to and expose some very serious topics like isolation race history and even stereotyping. Some families. isolation isn't a new concept people who were born with primary immune or pi disorders may have been living most of their lives this way and now dead grappling with watching the rest of the world experience isolation for the first time and everything that comes with that one amplify team members. Karen has a son that was born with severe combined immunodeficiency otherwise known as c. All bubble boy disease. Remember that movie from the seventies with the teenage john travolta playing a boy who lived his life in an actual bubble. Yeah that's actually a true story. Based on a boy with this disorder koreans family lived in complete isolation for the first three months of jude's life while they were waiting for him to have a life-saving bone marrow transplant. A common code. Could have killed him as he had no functioning white blood cells to fight off germs or infection. Every single day was filled with overwhelming. Feel while trying to keep it together for their oldest son. Shane who was seven at the time off the three months of living at louis children's hospital and the local ronald mcdonald house in while jude received chemotherapy and a stem cell transplant. Day will again in isolation for an entire year as he's new immune system grew and developed going through isolation again a second time this time along with the entire world has brought up all sorts of triggering memories and instances of ptsd for karan at her family. And you know what she's not the only one families off the immuno-compromised all over the world are dealing with these issues. While not be able to find the supplies they need to keep their homes clean all being terrified of going to the store for groceries and possibly bringing the virus home all while reading daily posts on social media about the virus being a hoax or a political issue used damage the reputation and image of other leaders. It's not just. Pi families that is experiencing different kind of fear in isolation. domestic violence is a terrifying issue. The world is always contending with and for those in unsafe situations at home. Isolation.

jude Shane severe combined immunodeficien washington john travolta leo cornell koreans ronald mcdonald house Karen ptsd karan
"severe combined immunodeficiency" Discussed on The BreakPoint Podcast

The BreakPoint Podcast

04:18 min | 2 years ago

"severe combined immunodeficiency" Discussed on The BreakPoint Podcast

"Before we can really celebrate breakthroughs in medical technology. We must know what is praiseworthy. And what is not? But the Colson center, I'm John Stonestreet. This is break point and the nineteen seventy six made for TV movie, the boy in the plastic bubble a then unknown John Travolta introduced Americans to a condition technically called x linked severe combined immunodeficiency or speaking of called it since the movie bubble boy disease children with bubble. Boy disease can't fight off infections like the rest of us can because their immune system doesn't work properly by Travolta's character. Mini who suffer from the condition live in sealed sterile enclosures that protect them from being exposed to the infections that could kill them in the movie Travolta's character somehow develops enough immunity to risk leaving the plastic bubble in search of true love, but many of his real world counterparts aren't so fortunate. At least they weren't a recent article in the New England Journal of medicine announced that doctors might have found a cure for bubble. Boy. Disease doctors at Saint Jude's Children's Research Hospital in Memphis used an inactivated form of HIV to introduce genetic changes into the patient's bone marrow cells, these changes fixed the bone marrow. So it starts doing its job pumping out all three types of immune cells seven infants with a condition, which is caused by a genetic defect were treated between two thousand sixteen and two thousand eighteen six of them were able to leave the hospital within four weeks and the seventh is apparently on the way to join them for parents of the estimated thirty to forty children born with condition each year. What happened in Memphis is nothing less than a miracle. Children who are virtually certain to die sometimes by the age of two. Now have a chance at life the same week the results at Saint Jude's were announced we learned that Isreaeli scientists had succeeded in manufacturing a three D printed heart made from human cells while it doesn't beat and is the size of a rabbit tart, it's a potential first step. And what was previously the stuff of science fiction, printing, human organs for use in transplants? See everyday twenty two people in the US alone die while waiting and organ transplant being able to manufacture them from human cells instead of just waiting for other people to die would save thousands. If not more lives in addition to giving hope to people suffering from life threatening illnesses. These stories offer a chance for us to discuss the right and wrong uses the biotechnology several times over the last few years. I've talked on break point about the pitfalls of technologies like crisper, the gene editing technology used by Chinese scientists to genetically modify to embryos last year. I called the criticism of the scientist faux outrage. Not because I approve to what he did. But because his critics, including those who invented crisper knew this would happen and still did nothing to prevent it. So how do we know which of these medical advances in biotechnology is moral in which is not where do we even be? Begin to draw these moral lines. Well, here's a suggestion. We have to distinguish between healing and enhancing. What happened in Memphis is definitely healing. The goal is to keep these infants from dying similarly attempts to manufacture organs for transplant seems rooted in a desire to heal the one could easily imagine a scenario in the near future in which printed parts outperform those were born with what then should we allow humans to upgrade see the use of human creativity? And engineered to heal keeps with our status as co creators with God. Obviously the difference between our creative powers and God's is literally infinite, but part of being created in God's image is the ability to use that creativity to care for creation, including ourselves in contrast when we use biotechnology to enhance humans even to create superhumans were attempting to usurp God as creator, we seek to create ourselves in our own image. Instead of conforming to. God's intentions for us. So far what happened in Memphis in Israel doesn't cross that line between healing and enhancement. But as fast as technology advances these days, we can never stop asking the question for breakpoint, I'm John Stonestreet.

Memphis John Travolta God John Stonestreet Saint Jude severe combined immunodeficien New England Journal of medicin US Research Hospital Disease scientist Isreaeli Israel four weeks
"severe combined immunodeficiency" Discussed on Probably Science

Probably Science

04:09 min | 2 years ago

"severe combined immunodeficiency" Discussed on Probably Science

"Anyone that had had open heart surgery in a in a you know in a procedure where they would have to stop your heart to do. Right. Would be legally dead, right? Like nineteen hundred hundred. Yeah. Nineteen o five definition that is dead that person is dead. But now, you had we kept the blood pumping through the body by official means and the and kept it is United and and that person woke up with no with the same brain functions as they did before out. Right. Yeah. Some tricky stuff. But for now, we're pretty sure I guess that these pigs were not temporaries didn't know. No, they this was a few brain cells. Let's not forget in the first place. They were killed to be in. So. We're already unethical weird territory Mets, mathematically, -tarian vegan. But we've decided that we can eat animals, and that sort of indisputable cultural like, no, these are we eat these take these living things, and we eat them. I don't feel great about it. Me neither there is another study, which is linked to his article that an implant is thought to speech, scientists developed a brain implant the people's minds into thoughts to speech, which the team at the UC. San Francisco say is exhilarating looks at the micro movements of like muscles related to forming words is it like before. Sometimes when you think in terms of actual words, you make you so contra hiding of the movements where you would be speaking. I think it might be close. Yes. This is a story that was only posted two hours ago and the BBC news websites. Okay. So so it's different. I'm checking it out as well. Let's let's jump onto a different story. Anyway, I think this is that reminds me vaguely of I think this might have been the thoroughness documentary about the guy who is talking about lie detector tests, and how they work. Yeah. There's sort of like morally coated like if you are like the test doesn't pick up whether you're lying it picks up. Whether you are doing something, you think is wrong. Right. So like if I thing. Yes, the dice thing for charity. So if basically like I this is a this is not the exact version of it. But like, I'm you have me hooked up to a lie detector, you've told me that whatever I roll on the dice like you're going to donate that many thousands of dollars to charity. Right. So I roll the dice, and I get to snake eyes. But I want you to donate twelve thousand dollars. So I say like, oh, this is too sticks. Is right. Lie detector test will not pick that up because my brain is like this is a moral decision. Right. You're doing a good thing by lime doing a good thing. Yeah. So they were saying like maybe Elizabeth homes in that same camp right now if she really believes that what she is doing is, right allied. That's definitely there are certainly compulsive lies who and pathological and psychopaths in general, people splaying psychopathy will generally beat light detectives. Because again, they. The action. Even if they even if they do have some fake more co where they are like, oh, no. I know what I'm doing is wrong. I also dug it shifts which is why it's like the t the test is like sort of misnamed it should be called like a discomfort. Detector. Yeah. I think OJ would totally pass lie detector at this point convinced himself of this alternate reality or me, if you ask like is it okay for you to be a geisha, right answers. The answer is definitely no. But I won't say that. US? Scientists say they've used HIV to make a gene therapy that cured eighth infants of severe combined immunodeficiency otherwise known as bubble boy disease. So the boy in the bubble of the film and the store various news stories. The idea these babies born little to no immune protection now have fully functional immune systems. Thanks to this research, develops in Tennessee that was published in the New England Journal of medicine sort of a hair the dog kinda thing. It's like, let's give let's go even farther. You don't have any immune system..

US severe combined immunodeficien official BBC New England Journal of medicin San Francisco United Tennessee HIV twelve thousand dollars two hours
"severe combined immunodeficiency" Discussed on KQED Radio

KQED Radio

05:45 min | 2 years ago

"severe combined immunodeficiency" Discussed on KQED Radio

"York Times national baseball writer Tyler Kettner, you talked to hundreds of pitchers about what they throw and how they try to get a mental edge over the hitters. They face. His new book is K a history of baseball in ten pitches. Join us. Rush air Thursday afternoon. One pm Thursday evening seven PM on K Q E D. I'm Marco werman each day. We give you the world. Into the center of the world is curious at total chaos in Venezuela right now. The world is surprising. These governments is completely the world is unexpected Japanese really have history today the world is right here. Join us. Join us Thursday afternoon beginning at two. Thirty years ago when he was a kid guy created a superhero. And now lo these many years, then I did is an Amazon is easy to the, hey, you're out it's easier to just the fate out because there's so much fatigue. I'm KAI Ryssdal. The tick, and it's returned to the small screen exam. Marketplace. Marketplace on Thursday afternoon at four PM. And since it's Thursday, we'll have political breakdown for you Thursday at six thirty pm with Maria logos. And Scott Shafer, they will talk with LA county. Labor federation, president rusty Hicks in LA, he represents some six hundred thousand workers. And now he is vying to become the chair of the California Democratic party, rusty Hicks, political breakdown again, Thursday evening, six thirty here on K Q E D public radio. From NPR news. It's all things considered. I'm Audie Cornish. Nelson Chang sometimes rare diseases allow for scientists to pioneer bold, new ideas. That's been the case with a condition that affects fewer than a hundred babies a year in the US, those babies are born without a functioning immune system and the quest to cure them as lead Vance's in technique called gene therapy NPR's, Richard Harris reports the disease is called severe combined immunodeficiency or skid met Portia Stanford. Pediatrician says you might be familiar with it. It was made famous I guess in the mid seventies. When the bubble boy was described in in documented documentary, and I think he captured the imagination of a lot of people David Vetter spent most of his short life in a plastic bubble to protect him from infection. He died at the age of twelve things have come a long way since then all babies born in the United States are now screened for this condition and standard treatment of bone. Marrow transplant succeeds more than ninety percent of the time when it's done promptly yet. Skit remains a source of great interest to researchers. This is one of those diseases in which there's probably more doctors and scientists studying the disease than patients who have the disease in the nineteen nineties European scientists actually cured it in some patients. Using gene therapy this technique involves removing defective. Blood cells from a patient inserting, a new gene with the help of a virus? And then putting this back into the body those cells than build up the patient immune systems, Dr Donald Kohn at UCLA says at first it looked really good at initially together. Twenty patients, they all had immune recovery. But over time five of them went on to develop leukemia. Scientists scramble to figure out how to inject new genes into cells without triggering that blood cancer, and they're cautiously optimistic they've succeeded since then there have been gradual improvements in the technique the latest advance involving eight infants shows that a short dose of chemotherapy. Maybe helped the new sales take root the infants ended up with apparently healthy immune systems. I am thrilled to CDs. Outstanding results Eveline among Sasha at Saint Jude's Children's Research Hospital in Memphis is first author of a paper reporting these findings in the New England Journal of medicine to be able to see these babies in my clinic now is told l'urss Israeli very rewarding. They live normal lives are not any different from my daughter's today most children with skid who get a bone marrow transplant also need ongoing treatment, including lifetime injections of antibodies. Jennifer puck a pediatrician at UC San Francisco and a study collaborator says infants got the newest gene therapy don't need that medication and they're growing. Normally they're getting cold like everybody else and they get over infection. So I would say that that is a cure. Of course, she adds that they'll be watched carefully for signs of leukemia. And to see if the effects of therapy are wearing off. In her mind. The key is finding these children early through newborn screening before they start to get life threatening infections that had typically been the case. And now we're seeing happy about little newborns who just look perfectly normal. This is not only good news for those rare families that disease provides a good opportunity for all those antics to develop even newer, gene. Therapy techniques, for example, instead of inserting a healthy, gene met Portius at Stanford. His used a powerful gene editing technique called crisper to correct the genetic error in skid blood cells. It works with human cells in a dish. And this really sets the stage then for testing the approach in a clinical trial. Hopefully in the next twelve to eighteen months all this makes the leukemia setback from the nineteen nineties feel like a fading memory Cohn at UCLA said for more than a decade. It seems that the field was a dead end. But no more just nice to see another success for gene therapy, Richard. Harris NPR news. It came to be known as v.

NPR leukemia Richard Harris UCLA United States rusty Hicks Marco werman KAI Ryssdal Audie Cornish Tyler Kettner Scott Shafer Amazon David Vetter LA county York Times severe combined immunodeficien writer
"severe combined immunodeficiency" Discussed on 90.3 KAZU

90.3 KAZU

04:36 min | 2 years ago

"severe combined immunodeficiency" Discussed on 90.3 KAZU

"Silly your childhood home can be an emotional transaction. Not least because the things you like about it might be a turnoff to buyers. Unfortunately, it seems like a lot of people these days they want a whole new house on the anti people went off the shelf ready on resolve the house, you grew up in and today's housing market next. Marketplace. Stay with us for marketplace. That's coming up at five thirty tonight right here on KABC. You the time now four fifty. From NPR news. It's all things considered. I'm Audie Cornish. Nelson chain sometimes rare diseases allow for scientists to pioneer bold, new ideas. That's been the case with a condition that affects fewer than a hundred babies a year in the US, those babies are born without a functioning immune system and the quest to cure them has led to advances in technique called gene therapy NPR's, Richard Harris reports the disease is called severe combined immunodeficiency or skid met Portia Stanford. Pediatrician says you might be familiar with it. It was made famous I guess in the mid seventies. When the bubble boy was described in in Documenta documentary. And I think he captured the imagination of a lot of people David Vetter spent most of his short life in a plastic bubble to protect him from infection. He died at the age of twelve things have come a long way since then all babies born in the United States are now screened for this. Edition and standard treatment a bone marrow transplant succeeds more than ninety percent of the time when it's done promptly yet. Skit remains a source of great interest to researchers. This is one of those diseases in which probably more doctors and scientists studying the disease than patients who have the disease in the nineteen nineties European scientists actually cured it in some patients. Using gene therapy this technique involves removing defective. Blood cells from a patient inserting, a new gene with the help of a virus? And then putting the cells back into the body, though cells than build up the patient's immune systems, Dr Donald Kohn at UCLA says at first it looked really good and initially twenty patients, they all had immune recovery. But over time five of them went to develop leukemia to scramble to figure out how to inject new genes into cells without triggering that blood cancer, and they're cautiously optimistic they've succeeded since then there have been gradual improvements in the technique the latest advance involving eight infant. Shows that a short dose of chemotherapy helped the new cells take root, the infants ended up with apparently healthy immune systems. I am thrilled to CDs. Outstanding results Eveline among Sasha at Saint Jude's Children's Research Hospital in Memphis is first author of a paper reporting these findings in the New England Journal of medicine to be able to see these babies in my clinic now as toddlers Israeli very rewarding they live normal lives. There are not any different from my daughter's today most children with skid who get a bone marrow transplant also need ongoing treatment, including lifetime injections of antibodies. Jennifer puck a pediatrician at UC San Francisco and a study collaborator says infants who got the newest gene therapy don't need that medication and they're growing. Normally they're getting cold like everybody else, and they take it over infection. So I would say that that is a cure. Of course, she adds that they'll be watched carefully for signs of leukemia and to see if the. Of the therapy are wearing off in her mind. The key is finding these children early through newborn screening before they start to get life threatening infections that had typically been the case. And now we're seeing happy bouncy little newborns. Just look perfectly normal. This is not only good news for those rare families the disease provides a good opportunity for all the scientists to develop even newer, gene therapy techniques, for example, instead of inserting a healthy, gene. Matt Portius at Stanford. His used a powerful gene editing technique called crisper to correct the genetic error in skid blood cells. It works with human cells in a dish. And this really sets the stage than for testing the approach in a clinical trial. Hopefully in the next twelve to eighteen months. All this makes the leukemia setback from the nineteen nineties feel like a fading memory Cohn at UCLA said for more than a decade. It seemed that the field was a dead end. But no more just nice to see. Another success for gene therapy. Richard Harris, NPR news. It came to be known as Chella..

David Vetter leukemia NPR UCLA Richard Harris United States Audie Cornish Matt Portius New England Journal of medicin Eveline severe combined immunodeficien Dr Donald Kohn Nelson Portia Stanford Cohn Jennifer puck
'Bubble Boy' Disease Helped By Advances In Gene Therapy

Fresh Air

03:54 min | 2 years ago

'Bubble Boy' Disease Helped By Advances In Gene Therapy

"Sometimes rare diseases allow for scientists to pioneer bold, new ideas. That's been the case with a condition that affects fewer than a hundred babies a year in the US, those babies are born without a functioning immune system and the quest to cure them has led to advances in technique called gene therapy NPR's, Richard Harris reports the disease is called severe combined immunodeficiency or skid met Portia Stanford. Pediatrician says you might be familiar with it. It was made famous I guess in the mid seventies. When the bubble boy was described in in Documenta documentary. And I think he captured the imagination of a lot of people David Vetter spent most of his short life in a plastic bubble to protect him from infection. He died at the age of twelve things have come a long way since then all babies born in the United States are now screened for this. Edition and standard treatment a bone marrow transplant succeeds more than ninety percent of the time when it's done promptly yet. Skit remains a source of great interest to researchers. This is one of those diseases in which probably more doctors and scientists studying the disease than patients who have the disease in the nineteen nineties European scientists actually cured it in some patients. Using gene therapy this technique involves removing defective. Blood cells from a patient inserting, a new gene with the help of a virus? And then putting the cells back into the body, though cells than build up the patient's immune systems, Dr Donald Kohn at UCLA says at first it looked really good and initially twenty patients, they all had immune recovery. But over time five of them went to develop leukemia to scramble to figure out how to inject new genes into cells without triggering that blood cancer, and they're cautiously optimistic they've succeeded since then there have been gradual improvements in the technique the latest advance involving eight infant. Shows that a short dose of chemotherapy helped the new cells take root, the infants ended up with apparently healthy immune systems. I am thrilled to CDs. Outstanding results Eveline among Sasha at Saint Jude's Children's Research Hospital in Memphis is first author of a paper reporting these findings in the New England Journal of medicine to be able to see these babies in my clinic now as toddlers Israeli very rewarding they live normal lives. There are not any different from my daughter's today most children with skid who get a bone marrow transplant also need ongoing treatment, including lifetime injections of antibodies. Jennifer puck a pediatrician at UC San Francisco and a study collaborator says infants who got the newest gene therapy don't need that medication and they're growing. Normally they're getting cold like everybody else, and they take it over infection. So I would say that that is a cure. Of course, she adds that they'll be watched carefully for signs of leukemia and to see if the. Of the therapy are wearing off in her mind. The key is finding these children early through newborn screening before they start to get life threatening infections that had typically been the case. And now we're seeing happy bouncy little newborns. Just look perfectly normal. This is not only good news for those rare families the disease provides a good opportunity for all the scientists to develop even newer, gene therapy techniques, for example, instead of inserting a healthy, gene. Matt Portius at Stanford. His used a powerful gene editing technique called crisper to correct the genetic error in skid blood cells. It works with human cells in a dish. And this really sets the stage than for testing the approach in a clinical trial. Hopefully in the next twelve to eighteen months. All this makes the leukemia setback from the nineteen nineties feel like a fading memory Cohn at UCLA said for more than a decade. It seemed that the field was a dead end. But no more just nice to see. Another success for

David Vetter Leukemia United States Ucla Severe Combined Immunodeficien New England Journal Of Medicin Matt Portius Portia Stanford Eveline NPR Dr Donald Kohn Richard Harris Jennifer Puck Cohn San Francisco Memphis Saint Jude Sasha
"severe combined immunodeficiency" Discussed on KCRW

KCRW

04:05 min | 2 years ago

"severe combined immunodeficiency" Discussed on KCRW

"Us updated. With the news four fifty case yard NPR news. It's all things considered. I'm Audie Cornish nine sometimes rare diseases allow for scientists to pioneer bold, new ideas. That's been the case with a condition that affects fewer than a hundred babies a year in the US, those babies are born without a functioning immune system and the quest to cure them as lead to advance in technique called. Gene therapy. NPR's Richard Harris reports the disease is called severe combined immunodeficiency or skid. Matt Portia Stanford. Pediatrician says you might be familiar with it. It was made famous I guess in the mid seventies. When the bubble boy was described in in Documenta documentary. And I think he captured the imagination of a lot of people David Vetter spent most of his short life in a plastic bubble to protect him from infection. He died at the age of twelve things have come a long way since then all babies born in the United States are now screened for this condition and standard treatment a bone marrow transplant succeeds more than ninety percent of the time when it's done promptly it. Skit remains a source of great interest to researchers. This is one of those diseases in which there's probably more doctors and scientists studying the disease than patients who have the disease in the nineteen nineties European scientists actually cured it in some patients. Using gene therapy this technique involves removing defective. Blood cell. From a patient inserting a new gene with the help of a virus? And then putting this back into the body those cells than build up the patient's immune systems, Dr Donald Kohn at UCLA says at I looked really good at initially. I think of the twenty patients they all had immune recovery. But over time five of them went on to develop leukemia. Scientists scramble to figure out how to inject new genes into cells without triggering that blood cancer, and they're cautiously optimistic they've succeeded since then there have been gradual improvements in the technique the latest advances involving eight infants shows that a short dose of chemotherapy helped the new cells take root, the infants ended up with apparently healthy immune systems. I am thrilled to CDs. Outstanding results Eveline among Sasha at Saint Jude's Children's Research Hospital in Memphis is first author of a paper reporting these findings in the New England Journal of medicine to be able to see these babies in my clinic now as toddlers Israeli. Very rewarding. They live normal lives are not any different from my daughter's today most children with skid who get a bone marrow transplant also need ongoing treatment, including lifetime injections of antibodies. Jennifer puck a pediatrician at UC San Francisco and study collaborator says infants who got the newest gene therapy don't need that medication and they're growing. Normally they're getting cold like everybody else and they take over infection. So I would say that that is a cure. Of course, she adds that they'll be watched carefully for signs of leukemia and to see if the effects of the therapy are wearing off in her mind. The key is finding these children early through newborn screening before they start to get life threatening infections that had typically been the case and now are seeing happy bouncy little newborns who just look perfectly normal. This is not only good news for those rare families that disease provides a good opportunity for all the scientists to divert. Philip even newer, gene therapy techniques, for example, instead of inserting a healthy, gene. Matt Portia at Stanford has used a powerful, gene editing technique called crisper to correct the genetic error in skid blood cells. It works with human cells in a dish. And this really sets the stage then for testing the approach in a clinical trial. Hopefully in the next twelve to eighteen months all this makes the leukemia setback from the nineteen nineties feel like a fading memory Cohn at UCLA said for more than a decade. It seems that the field was a dead end. But no more nice to see another success for gene therapy. Richard Harris, NPR news. It came to be known as.

NPR David Vetter leukemia Richard Harris United States UCLA Matt Portia Stanford Audie Cornish severe combined immunodeficien Eveline New England Journal of medicin Matt Portia Dr Donald Kohn Jennifer puck Cohn Stanford San Francisco
"severe combined immunodeficiency" Discussed on KQED Radio

KQED Radio

04:40 min | 2 years ago

"severe combined immunodeficiency" Discussed on KQED Radio

"An estimated twenty five thousand of them are in the San Francisco Bay area here from some of the bay area's meeting voices on issues surrounding homelessness. Meena Kim leads a panel of experts to discuss the state of the crisis. How we got here where we're headed next tonight at eight PM on. Real person fiction has got millions of people reading and talking. I'm Joshua Johnson. The impact of fan fiction about real people next time on one A B with us for one a tonight at eleven here on kqed public radio. From NPR news. It's all things considered. I'm Audie Cornish Chang. Sometimes rare diseases allow for scientists to pioneer bold, new ideas. That's been the case with a condition that affects fewer than a hundred babies a year in the US, those babies are born without a functioning immune system and the quest to cure them has led to advance in technique called gene therapy NPR's, Richard Harris reports the disease is called severe combined immunodeficiency or skid met Portia Stanford. Pediatrician says you might be familiar with it. It was made famous I guess in the mid seventies. When the bubble boy was described in documented documentary, and I think he captured the imagination of a lot of people David Vetter spent most of his short life in a plastic bubble to protect him from infection. He died at the age of twelve things have come a long way since then all babies born in the United States are now screened for this condition and. Standard treatment of bone marrow transplant succeeds more than ninety percent of the time when it's done promptly yet. Skit remains a source of great interest to researchers. This is one of those diseases in which there's probably more doctors and scientists studying the disease than patients who have the disease in the nineteen nineties European scientists actually cured it in some patients. Using gene therapy this technique involves removing defective. Blood cells from a patient inserting, a new gene with the help of a virus? And then putting the cells back into the body those cells than build up the patient immune systems, Dr Donald Kohn at UCLA says at first it looked really good at initially together. Twenty patients, they all had immune recovery. But over time five of them went to develop leukemia. Scientists scramble to figure out how to inject new genes into cells without triggering that blood cancer, and they're cautiously optimistic they've succeeded since then there have been gradual improvements in the technique the latest advance involving eight infants shows that. Short dose of chemotherapy, helped the new cells take root, the infants ended up with apparently healthy immune systems. I am thrilled to see outstanding results. Eveline among Sasha at Saint Jude's Children's Research Hospital in Memphis is first author of a paper reporting these findings in the New England Journal of medicine to be able to see these babies in my clinic now as toddlers Israeli very rewarding. They live normal lives are not any different from my daughter's today most children with skid who get a bone marrow transplant also need ongoing treatment, including lifetime injections of antibodies. Jennifer puck a pediatrician at UC San Francisco and a study collaborator says infants who got the newest gene therapy don't need that medication and they're growing. Normally they're getting cold like everybody else. Take it over infection. So I would say that that is a cure. Of course, she adds that they'll be watched carefully for signs of leukemia and to see if the effects of the therapy are wearing off in her mind. The key is finding these children early through newborn screening before they start to get life threatening infections that had typically been the case. And now we're seeing happy bouncing little newborns who just look perfectly normal. This is not only good news for those rare families the disease provides a good opportunity for all the scientists to develop even newer, gene therapy techniques, for example, instead of inserting a healthy, gene. Matt Portia at Stanford. His used a powerful gene editing technique called crisper to correct the genetic error in skid blood cells. It works with human cells in a dish. And this really sets the stage then for testing the approach in a clinical trial. Hopefully in the next twelve to eighteen months all this makes the leukemia setback from the nineteen nineties. Feel like a fading memory Cohn at UCLA said for more than a decade. It seems that the field was dead end. But no more nice to see another success for gene therapy. Richard Harris, NPR news. It came to be known as.

leukemia NPR David Vetter UCLA Richard Harris United States San Francisco Bay Matt Portia Joshua Johnson Meena Kim Audie Cornish Chang Eveline severe combined immunodeficien Portia Stanford New England Journal of medicin kqed Dr Donald Kohn
"severe combined immunodeficiency" Discussed on 90.3 KAZU

90.3 KAZU

03:52 min | 2 years ago

"severe combined immunodeficiency" Discussed on 90.3 KAZU

"It's all things considered from NPR news. I'm elsa. Audie. Cornish. Coming up historic flooding in Nebraska last month. Devastated small town. Now, some people are considering leaving. None of these towns can stand to lose anybody. It's gonna be a challenge checking in on the recovery in Nebraska that story after these news headlines on all things considered. Live from NPR news in Culver City, California, Dwayne Brown and office to protect whistleblowers at the department of veterans affairs is under investigation for retaliation against whistleblowers. NPR's quil Lawrence has the story. An NPR investigation found that the VA has the highest rate of whistleblower complaints in the federal government. A new office created by congress the office of accountability. And whistleblower protection was supposed to help shield employees from Italian when they reported waste or abuse at VA branches the office in question got off to a rough start last year when its director refused to provide information to the VA's inspector general now, the is looking into complaints that this new office has been reporting whistleblowers back to their local supervisors several told NPR that this has resulted in professional retaliation the office would not give details about an ongoing investigation. Quil Lawrence NPR news. In paris. The French prime minister is announced an international competition for architects to replace the spire of Notre Dame cathedral destroyed in this week's fire. NPR's ofeibea Quist arcton reports the government has been planning it's focus strategy and response to the disaster special. French cabinet meeting was dedicated to the reconstruction of knuckle them off to which prime minister Philippe outlined a global competition to design a new spot for the cathedral. He said this was to give them a spot. Most you to more modern techniques and challenges the French prime minister says the competition for architects encourages dialogue, asking should know. Cut. Them's original spa, be recreated all updated with a MO modern design the world watched in horror as the nineteenth century spa came crashing down Monday. When parts of the cathedral went up in flames contributions from around the world are reportedly approaching nearly a billion dollars for the restoration of not for them affair be Akwa. Stockton NPR. News Paris stocks finished, slightly lower on Wall Street. This is NPR. Scientists continue to improve an approach to a medical treatment. Call gene therapy as NPR's Richard Harris tells us the latest refinement involves a rare disease in which children are born without functioning immune systems, the disease is called severe combined immunodeficiency, but it's better known as the bubble boy disease after a child who in the nineteen seventies lived in plastic containment to be protected from germs in the decade since scientists have steadily improved treatment for this incredibly rare disease. Now, scientists from Saint Jude's Children's Research Hospital report that the latest strategy has left eight of its young patients at least for now in good health with fully functioning immune systems. Their strategy is similar to one previously approved for use in Europe. These advances are helping scientists develop more effective techniques for other kinds of gene therapy treatments as well. Richard Harris, NPR news. They Martha Stewart brand of cookware magazines towels is changing corporate hands again Stewart, we'll still oversee the company which is being sold to marquee brands were about one hundred seventy five million dollars. Stewart recently struck a deal to make products containing CBD compound derived from him that doesn't cause a high as part of the deal marquee will also acquire the brand of TV chef emerald legacy marquee already owned several clothing and footwear brands, including Ben Sherman and body glove. Stocks finished slightly lower on Wall Street as a drop in healthcare shares overshadowed others. This is NPR..

NPR Lawrence NPR prime minister Martha Stewart paris Nebraska VA Richard Harris Notre Dame cathedral severe combined immunodeficien quil Lawrence Culver City Philippe Europe ofeibea Quist congress
"severe combined immunodeficiency" Discussed on NEJM This Week - Audio Summaries

NEJM This Week - Audio Summaries

02:02 min | 2 years ago

"severe combined immunodeficiency" Discussed on NEJM This Week - Audio Summaries

"PCI who do not have high risk, clinical or angiographic features or a high risk of bleeding early. Omission of aspirin therapy and treatment with a direct oral anticoagulant plus clopidogrel is entirely warranted. However in patients undergoing complex multi vessel or high risk PCI or. In those presenting with high risk acute coronary syndrome, aspirin should probably not be routinely omitted for at least several weeks or longer depending on bleeding risk, although guideline committees will now have to grapple with incorporating the results of this trial into specific recommendations, it is clear that a one-size-fits-all policy is unlikely to apply in these patients. Lenti viral, gene therapy combined with low dose byu south N in infants with skid x one by Evellina mom cars from Saint Jude's Children's Research Hospital. Memphis, Tennessee, allogeneic Jimeta poetic stem cell transplantation, for x linked severe combined immunodeficiency skid x one often fails to reconstitute immunity associated with t cells b cells and natural killer cells. When matched sibling donors are unavailable. Unless high dose chemotherapy is given these investigators performed phase one to safety and efficacy study of a Lenten viral vector to transfer I l to RG complementary DNA to bone marrow stem cells after low exposure targeted buechele fan conditioning in eight infants with newly diagnosed skid x one. Bone marrow. Harvest bue self end conditioning and sell infusion had no unexpected side effects in seven infants the numbers of CD three CD four and naive. Cd?.

aspirin severe combined immunodeficien acute coronary syndrome clopidogrel Memphis Evellina Tennessee Research Hospital Jimeta
"severe combined immunodeficiency" Discussed on The Meb Faber Show

The Meb Faber Show

03:56 min | 2 years ago

"severe combined immunodeficiency" Discussed on The Meb Faber Show

"I would have to diversify my personal portfolio away from the public markets. I was seeking to generate alpha from uncollated asset class, but at the same time I wanted to invest in things that I cared about. Or at a minimum. I felt could have some sort of positive impact on those around me. So it was from financial perspective. It was important for me to gain exposure to these potential positive black swan events and build into my portfolio, the concept of positive optionality. So pretty listen to who may be unfamiliar with that term positive optionality means there's symmetric risk for ward with a positive steward the recited question. So in other words, you have the chance to make more than you lose on any given investment or any given trait. Another way to think about that sort of a low cost mistake with known maximum losses and the Patel. Title unbounded, upside pale, that's basically sort of a central feature of positive black swan. So I was trying to think how do I engineer my portfolio to take advantage of that? And that's how I got into these sort of VC style investments that we do at by over and that's part of a well diversified portfolio. So as I mentioned, I've been investing since high school what a diversified portfolio, Iran business development at Serm, and it was really sermon. I was there for about five plus years. There's really at Serm where I saw firsthand the shoot impact and investing in some of these cutting edge healthcare technologies had and how much just really captivated inspired literally. Like, everyone I talked to about the types of technologies we funded at some did you talk a little bit about your time at, sir. Maybe to the extent you can walk through a little bit about some of the projects you worked on or some of the way that it was structured chose fabulous place. I think this really drives home the point of the types of technologies that we invested in at some. So there's a pretty rare disease called severe combined immunodeficiency and the very small percentage of children are born with this disease. It's a genetic disease. If any our listeners are Seinfeld fans they've done it episode with the boy in the bubble bubble boy disease that is skid children born with its disease. They have one copy of Jean that doesn't work properly. So they're entire union system doesn't function, and as you can imagine they can't live normal life. They're subjected to frequent infections hospital visits typically died at a relatively early age. There's one little girl in particular. I became close with during my time at Serm her name is Evy, and she was actually sure of this disease from some of the work that we funded at Serm. The technology was inside of UCLA at the time. The technology was subsequently spun out into a company the biotech company that went public and now thirty kids have been cured of the disease severe combined immunodeficiency. And so to me, that's just really sp-. Firing story. And that's the kind of stuff that we funded at syrup, and there are lots of examples of funding that type of cutting edge orc, particularly in academia or in small biotech companies and that work has a measurable impact. And by the way, those companies are doing well, some of those companies have gone public. Right. So that investors making money off of that. So it was really at Serm where I learned that the institutional model of financing and investing in this cutting healthcare could be applied to the retail sector and just so you don't think that example of every is one off type thing right less than five percent of diseases caused by a single gene defect or what we call mono genyk diseases have any sort of available treatment today. So that's a lot of the stuff that we did at some. And so what really drove me to found by overed was again sort of looking at how do you build a well diversified portfolio? How do you gain access to some of these high grow high impact investments where you can have the potential to generate a great return? And so if you look at the Merican association of individual investors, data the average retail investors portfolio consists of about thirty two percent individual stocks thirty six percent stock funds..

Serm severe combined immunodeficien Evy engineer ward UCLA Iran Merican association overed Seinfeld Jean thirty six percent thirty two percent five percent
"severe combined immunodeficiency" Discussed on WBZ NewsRadio 1030

WBZ NewsRadio 1030

03:28 min | 2 years ago

"severe combined immunodeficiency" Discussed on WBZ NewsRadio 1030

"The government is open again at least for the next three weeks. His siblings Dettori spas, gangster, James Whitey Bulger are laying claim to his estate. A measles outbreak kits. The Pacific northwest. The New England Patriots are inviting fans to a Super Bowl send-off rally at Gillette stadium tomorrow morning at ten o'clock. He's a baby boy without an immune system and efforts are underway to help pay for his extensive medical treatments. WBZ's Doug cope tells us about the challenges at Taunton family is facing. Amanda acorn. Sunshine was born last November and has since suffered from what's known as severe combined. Immunodeficiency acorn says Shane's condition is tough on the family. You can't go in any stores. He can't go anywhere. Really our families can't come over. He's just stuck in the house, and we have to go to a store the kids. Stay in the car was one of us. And then the next one of us will go on the store afterwards. But we can't go anywhere family. Shane needs a bone marrow transplant and we'll be hospitalized for an extended period of time. So a gofundme page has been set up and fundraisers will be held at health clubs and Foxborough and north Attleboro Doug cope WBZ Boston's NewsRadio. The woman who heard three year old Casey Hathaway crying in the woods and tipped off searchers telling her story now the missing toddler was found alive in North Carolina on Thursday. Linda fraker says she was out walking her three dogs when she first heard a small cry. She says she took the dogs home. Then we're not to listen again after she heard the cry again notified a deputy looking out for that kid. There's no doubt in my mind looking out for that baby Hathaway disappeared from his great grandmother's home in craven county on Tuesday. Authorities say he was found about a quarter mile from that home and was wet and a little scratched up. But in good spirits looks like the NBA may be seeking a fare hike. Possibly this summer. This'll be the fourth increase since two thousand twelve though the first in three years the tea has been boosting fares more frequently, but it smaller rates to keep up with rising costs the process of raising fares is expected to start this Monday at the meeting of the agency's governing. Board a Connecticut us baseball coaches arrested at a Warwick Rhode Island hotel for allegedly soliciting child over the internet. Rhode Island state police say thirty three year old Christopher merchant of Canterbury. Connecticut use the internet to solicit a four. Eighteen year old boy to engage in sexual activity at the hotel merchant is a vice president and coach with the Plainfield little league in Plainfield Connecticut. Police say currently there is no evidence of any sexual involvement with members the Plainfield little league his arrest stems from an investigation by members of the Rhode Island internet crimes against children tax force Main's timeline for getting legalize marijuana. In stores is now uncertain and wake of the cancellation of a consulting deal to write rules main had awarded a two hundred thousand dollars bid to Los Angeles consultant biotech to help craft the rules about issues such as sales and packaging officials with through the bed after another bidder. Appealed main officials originally hoped the rules on legalize marijuana would be ready by April. Some changes are on the way YouTube ABC's, Dave Schreiber explains. The way some videos are promoted will change YouTube says it will stop promoting videos supporting sham science and other misinformation such as flat earth, videos and other. Conspiracy theories YouTube announced it will stop suggesting material it deems harmful or borderline content..

Shane Linda fraker James Whitey Bulger Taunton family YouTube Casey Hathaway Plainfield little league Pacific northwest Rhode Island government Doug cope Dettori Immunodeficiency Amanda acorn marijuana Warwick Rhode Island New England Patriots Gillette stadium Connecticut Foxborough
"severe combined immunodeficiency" Discussed on Health Care Rounds

Health Care Rounds

08:00 min | 3 years ago

"severe combined immunodeficiency" Discussed on Health Care Rounds

"Solution to rising drug costs as we've written. It's unlikely that CMS will be negotiating part d drug prices anytime soon. In may when the Trump administration announced its blueprint to lower drug prices, one of the initiatives. Four pillars was to develop incentives for manufacturers to lower list. Prices having them list drug prices on ads and publicly shaming them when they don't is one such incentive. But here's the thing. No one pays the list. Price for drugs. Drugs wack isn't the same as the average manufacturer price, which is the average of prices actually paid by wholesalers and retailers who by drugs for manufacturers. In addition, the average sales price, what manufacturers realized after discounts and rebates is far lower than list price, and the actual price paid by consumers varies widely depending on their insurance coverage or lack thereof. Glancing at the list of forty six novel drug approvals by the FDA in two thousand eighteen we've found that nearly every drug approved was cancer related or for a rare disease. This undoubtedly means that most of the drugs approve this year or expensive. This is no accident drugmakers for at least a decade have moved away from developing drugs that could be used by wide swaths of patients. Think blood pressure drugs cholesterol, lowering drugs antibiotics, one antibiotic was approved this year, instead manufacturers have focused on cancer drugs and rare disease treatments, which can fetch ten thousand dollars per month or more a cynic would say it's all about profit drug innovators can charge whatever they want to recoup their investments in research and development smaller patient populations naturally lead to higher prices supply and demand viewed another way the drug industry is behaving. Rationally there are plenty of safe and effective choices to lower your. Asteroid or blood pressure. But cancer kills two hundred seventy five thousand Americans each year. And there aren't many treatments for Dravid syndrome or dean assigned Yemen as severe combined immunodeficiency so pharma is putting its resources where they're needed most. We'll shaming pharma into lowering prices work. Probably not law. Consumers will be informed. As Verma says, they won't have all the information about what the drug will actually cost them. Our sense is most people will eventually ignore prices in DC ads. Just like they've learned to ignore the long and often scary list of possible side effects trailing every TV commercial. Here's what else you need to know. United health group will soon be launching its own electric health record. According to see you David which Mond on a quarterly earnings conference. Call which Mund said, quote, we will soon be releasing at scale a first of its kind fully integrated and fully portable individual health record that delivers personalized next best health actions to people and their caregivers and quote. He offered few details. Other than the company would leverage its mobile wellness platform rally to eventually reach. It's fifty million members also unclear is how United's platform would interact with existing popular electronic health record systems such as epic answer. CMS said that healthcare premiums on the federal health exchange are declining for the first time since the program was implemented in twenty fourteen. The average premium for the second lowest costs over plans for the twenty nineteen coverage will decline by one point five percent CMS said that since two thousand fourteen average individual market premiums on healthcare dot gov. More than doubled from two thousand seven hundred eighty four dollars per year in twenty thirteen to five thousand seven hundred and twelve dollars in two thousand seventeen factors contributing to the lower premiums include slower growth in some medical expenses and overpricing of plants in twenty seventeen less political uncertainty and insures growing familiarity with the market separately. CMS announce the launch of its bundled payment for care improvement advanced model which began on October first CMS said eight hundred and thirty two acute care hospitals and seven hundred fifteen physician group practices are participating in the program under BPA. There are thirty two bundled clinical. Episodes twenty nine impatient. And three outpatient participants are provided target prices before the start of each model year. Participants earn more if the cost for the episode are less than the target, but must repay Medicare. If the cost of the episode exceed the target. Walgreens in grand block Michigan-based McLaren healthcare announced a new strategic collaboration for health and pharmacy services. Under the agreement. Maclaren will offer a variety of healthcare services in Walgreens stores across Michigan, including retail healthcare clinics. Urgent care centers and other primary care offerings. Walgreens has acquired McLaren's pharmacy assets and will operate on site pharmacies at select McLaren locations. Mclaren health plan members will be able to access Walgreens pharmacy services at all locations. Presumably under a preferred agreement. President Trump signed to bipartisan bills into law that would ban pharmacy gag clauses at the pharmacy counter. The bills known as the patient right to know act and the no the lowest price act will allow pharmacists to discuss whether prescription is less expensive using insurance or paying out of pocket. The law affecting commercial insurance contracts takes effect immediately the law affecting Medicare beneficiaries takes effect January first two thousand twenty. A new national association of a CEO survey of ACO executives reveals deep opposition to see messes. Recently proposed changes to the Medicare program under its pathways to success proposed rule Seamus would allow an ACO to remain in upside only or track one risk model for two years. The proposed rule also would limit track. One ACS to twenty five percent of eligible shared savings compared with the current fifty percent rate. Overall, sixty one percent of the survey respondents oppose the proposed rule the largest area of opposition was the reduced shared savings rate for track. One ACO's with fifty seven percent in opposition more than a third or thirty six percent said they would be unlikely to continue under the revised policies. Walgreens stepped up its partnership with Labcorp to open six hundred testing centers inside Walgreens retail locations over the next four years. The agreement is an expansion of the seventeenth store pilot program that started in June twenty seventeen Stefanopulos sina CEO of Walgreen's, boots alliance said in a statement, quote, this reflects our commitment to transform our stores into neighborhood health destinations that provided differentiated consumer focused experience while providing access to a broad range of affordable healthcare services at a trusted and convenient setting. The dignity health C H I merger is one step closer to being finalized. After receiving the go ahead from the Vatican. The health systems also announced their board of trustees members last week, which will include six existing board members from each health system, the two currency ios. Kevin Lofton and Lloyd dean and an additional member to be determined. After alignment has been completed. The merger remains subject to state and other regulatory approvals. A medicine Inc. Signed a definitive agreement to acquire compassionate care. Hospice or C C H last Wednesday for three hundred and forty million Parsippany New Jersey-based C C H operates fifty three

Walgreens Medicare McLaren CEO United health group severe combined immunodeficien McLaren healthcare Yemen FDA ACO Verma Maclaren President Trump Mund Michigan United
"severe combined immunodeficiency" Discussed on Important, Not Important

Important, Not Important

04:32 min | 3 years ago

"severe combined immunodeficiency" Discussed on Important, Not Important

"In this kind of gets down to the key. The key step, which is how you actually modify the selves because it may if you can modify the immune cell to be meal, even. Better or to be able to make more copies of itself more efficiently in the body. Then then maybe you don't have to expand it as long outside the body. And then now the patient doesn't have to wait two weeks. They only need to wait a day or a couple of days or while that's that's a game changer with the the real game changer would be if we could you take cells out, modify them and put them back in, you know, on the course of an hour or two, and that's something as I know it's early. But I mean, is that theoretically something you guys are reaching for? I mean, that'd be incredible. Certain, certainly, when you think about you what one of the big the big barriers to these therapies are their cost in their complexity in. So that's that's something that we think about. You know, it's not necessarily something that you we weren't. We're working on right now in the lab, but route of the guiding. And you know, kind of ethos for our work, which is mainly center on how do we modify. T. cells in a easier, simpler, better way. If we can do it Neil in its the process is simpler than that means that the actual when we, you know, move into a clinical therapies that it can be shorter process in, hopefully cheaper one is well, what's a t. cell. Yeah. So we've been using. We've been saying immune cells throughout. There's a a lot of different types. One of the main ones and the most important one. So is this called a t cell? The t. cell is classically the cell type that will search for viral infections and tried to eliminate any cells that have been infected with a virus. Oh, all right. And so you can see. So you know, my pardon go, it's. If you also say, if you don't have t. cells, then you end up having a condition called a skit or severe combined immunodeficiency, which, but more commonly known as a bubble boy syndrome, right? So yeah. So without without these immune cells, you essentially you're, you're powerless to fight off a most infections that have evolved to infect humans numb. So, but but this has that has an important connection with the the cancer side of things which is at because t. cells are made to recognize our own cells, but our own cells that aren't healthy, our own cells that have been changed in some way change that they evolved recognize is a cell that was infected by a virus. But it turns out that they also can recognize cells that have been changed in a very different way which is cells that have become cancerous. Interesting. So the actual, you know. The systems though the process he said they use are very similar between these two and obviously there's differences. You know, I, I can imagine a somebody worked in the field listening to me, say that in Scoffing and writing down the big list of all the different. They're not here, but. Realized that was just. But, but these t. cells kind of have they have this inherent ability to look at our own cells and tell if there's something wrong, it's pretty dope, and that's in. That's the in. That's the kind of the backbone for what we want to build on top of them. And so you know, there's in the lab in in in what we studying, what many other labs study are ways that you know, this process doesn't work a hundred percent of the time, and there are there are ways that cancer can get around this process. Just like there's ways that viruses have evolved to get around the immune system right cancers do the same thing on the on this much more accelerated time scale, which is pretty scary to think about like your own cells, evolving inside your body to evade your own immune system. Yeah, what the ship may have page here, but cool, man. So what so what is, what is your big innovation? Say, explain this to us and why. Why? Why it's such a revolution? Well, I'll let time tell whether it's a revolution or not. We're we're excited about it the, like I said, the key..

Neil severe combined immunodeficien hundred percent two weeks
"severe combined immunodeficiency" Discussed on Two Scientists Walk Into a Bar

Two Scientists Walk Into a Bar

01:46 min | 3 years ago

"severe combined immunodeficiency" Discussed on Two Scientists Walk Into a Bar

"There are patients that are born with no natural killer cells and these patients suffer from very severe or disseminated viral infections such as herpes viral infections, chicken pox, pneumonia and about a fifth of these individuals, actually develop cancers that are related to viral infections. And then in terms of the adaptive immune response genetic deficiencies that are associated with diseases upped ability as absolute such a skit syndrome, so severe combined immunodeficiency these children have not cells or B cells, and they get very severe infection during the first year of life of severe bacterial violent fungal infections. And if they're. Left untreated. They typically will when die in the first year of life. So a good example actually, the bubble. Boy, he had a defect in one of the proteins that are required for a t and b cell development. But then they're also acquired deficiencies so HIV a great example where upon infection infected individuals get a deficiency of the T cell compartment. Conversely, you can get B-cell immunodeficiencies. There is an enzyme. That's called Bruton's tyrosine kinase. This enzyme is very critical for the development of B cells. And there are males that are born with defects or mutations into BT K Zayn that have no b cells, and hence, no plasma cells, and no antibody and these individuals need to be treated either with lifelong monthly gamma, a gamma globulin replacement or in more severe cases with..

severe combined immunodeficien Bruton pneumonia BT