Podcasts about evrysdi

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world.Novo's semaglutide shows promise in curbing alcohol use disorder, according to data published in JAMA Psychiatry. This supports the use of GLP-1 receptor agonists for addictive disorders. Meanwhile, the Trump administration is considering job cuts at the FDA. This could potentially lead to uncertainty and delays in regulatory reviews.In other news, Sanofi is facing a $250 million impairment after scrapping a J&J-partnered E. coli vaccine. Roche's Evrysdi has become the first pill cleared for spinal muscular atrophy. Pfizer has expanded the label for Adcetris to include non-Hodgkin lymphomas.Additionally, Newleos Therapeutics has raised $93.5 million to advance neuropsychiatric drugs from Roche's pipeline. All this information was shared by BioSpace, a platform that provides updates on the biotech and pharmaceutical industry.

Study identifies potential treatment strategy for high-threshold peanut allergy; Emblaveo approved for complicated intra-abdominal infections; new tablet formulation of Evrysdi; FDA fast tracks investigational agents for Alzheimer disease and cannabis use disorder.  

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world. In a recent report on the current trends and challenges in drug discount management (DDM) within the biopharma industry, economic uncertainty and revenue leakage are highlighted as major challenges. With downward pricing pressure, pharmaceutical companies are finding it difficult to maintain profitability. The report also discusses technology solutions that can make DDM easier for biopharma companies.Edwards Lifesciences has received CE Mark approval for its tricuspid valve replacement, allowing the company to market the device in Europe. The company expects to gain approval from the US Food and Drug Administration (FDA) late next year. Meanwhile, Interventional Systems has received FDA clearance for its miniature robot, the Micromate system, which can be used with CT navigation in procedures such as biopsies and ablations. Supply disruptions are delaying surgeries and compromising patient care, according to a survey. A patient safety nonprofit has called for long-term, nationally coordinated solutions to address persistent shortages. The FDA has finalized COVID-era guidance that extends flexibility for some remote monitoring devices. Abbott Laboratories has raised its outlook after a strong Q3 performance. Sales of its Freestyle Libre continuous glucose monitor rose nearly 31%. Pfizer has priced its COVID drug Paxlovid at $1,400 for a five-day course.Henry Ford Health and Ascension have announced a joint venture to create a $10.5 billion healthcare system. Senators from the Senate Veterans Affairs Committee have urged the CMS to rescind or revise its staffing proposal for nursing facilities. Washington hospitals are suing the state over an expanded interpretation of a charity care law. Walgreens and Alignment Healthcare are partnering on co-branded Medicare Advantage plans. Elevance Health could face a $500 million revenue hit in 2025 due to a drop in Medicare Advantage star ratings.Roche has reported lower third-quarter sales due to the impact of the contracting COVID-19 market. Intellia Therapeutics has received phase III approval from the FDA for its gene editing therapy. Royalty Pharma has provided an additional $1 billion for royalties on Roche's Evrysdi. UCB has also received FDA approval for its plaque psoriasis therapy, Bimzelx.Beam is planning to lay off 100 employees and seek partners in a research restructuring. PTC Therapeutics has announced a $1 billion deal with Royalty Pharma to sell the spinal muscular atrophy drug royalties for its therapy Evrysdi. Roche has reported growing sales of its eye drug Vabysmo in the US market. Ultragenyx plans to spin out a new company focused on gene therapy for Alzheimer's disease.The article discusses various topics related to marketing and advertising. General Mills has refreshed its fruit snack brands, such as Gushers, in an effort to stand out in the social media age. Inspire Brands prioritizes

Kyle Filkins and Kris Napper were both born with SMA, spinal muscular atrophy. Meeting as kids at The Muscular Dystrophy Association summer camp, they have developed a friendship and a documentary. After beginning the use of the drug Evrysdi, their rare genetic disease stopped progressing, and the oral form of this medication allowed them to avoid spinal injections. Now in their thirties, Kris and Kyle are advocating for the community of those with SMA, working on their documentary, and looking forward to more adventures.  Kris explains, "Just shortly before Evrysdi was released and made available, there was another drug called Spinraza that was the first treatment ever for our disability. It was a spinal injection, which poses some complications as far as it's a procedure with some risk. It can be painful, and there's the risk of hitting nerves and causing damage. It's not the most fun thing to go through, but it was beneficial. So I was doing that." "So for young children, just being diagnosed can be life-changing because you can stop that progression before it really hits. For us, we've already had significant progression. Still, hopefully, this will stop it where it is, and we'll be able to continue our lives for the foreseeable future, a lot better and longer than originally anticipated." Kyle elaborates, "Before I was on any type of therapy, I would go to bed one day able to do something, and I'd wake up the day after not being able to do that. And it would never come back. And it's pretty great to be able to go to bed and sleep securely knowing that what I did today I can, for the most part, probably do tomorrow. It's definitely life-changing. That's for sure." "There's been a lot of people interested in this story. Kind of piggybacking here on what Napper said, at the outset, it was really kind of just a budget film and like, "Oh, maybe they'll do cool activities and make jokes and stuff." But then with the advent of this therapy and specifically Evrysdi, that's really changed the outlook of the scope of the film, and for the better, as Napper said."  @KKtheFilm #SMA #SpinalMuscularAtrophy #MuscularDystrophy #RareDisease #Evrysdi  KrisandKyletheFilm.com SnappyClothing.com Evrysdi.com Download the transcript here

Kyle Filkins and Kris Napper were both born with SMA, spinal muscular atrophy. Meeting as kids at The Muscular Dystrophy Association summer camp, they have developed a friendship and a documentary. After beginning the use of the drug Evrysdi, their rare genetic disease stopped progressing, and the oral form of this medication allowed them to avoid spinal injections. Now in their thirties, Kris and Kyle are advocating for the community of those with SMA, working on their documentary, and looking forward to more adventures.  Kris explains, "Just shortly before Evrysdi was released and made available, there was another drug called Spinraza that was the first treatment ever for our disability. It was a spinal injection, which poses some complications as far as it's a procedure with some risk. It can be painful, and there's the risk of hitting nerves and causing damage. It's not the most fun thing to go through, but it was beneficial. So I was doing that." "So for young children, just being diagnosed can be life-changing because you can stop that progression before it really hits. For us, we've already had significant progression. Still, hopefully, this will stop it where it is, and we'll be able to continue our lives for the foreseeable future, a lot better and longer than originally anticipated." Kyle elaborates, "Before I was on any type of therapy, I would go to bed one day able to do something, and I'd wake up the day after not being able to do that. And it would never come back. And it's pretty great to be able to go to bed and sleep securely knowing that what I did today I can, for the most part, probably do tomorrow. It's definitely life-changing. That's for sure." "There's been a lot of people interested in this story. Kind of piggybacking here on what Napper said, at the outset, it was really kind of just a budget film and like, "Oh, maybe they'll do cool activities and make jokes and stuff." But then with the advent of this therapy and specifically Evrysdi, that's really changed the outlook of the scope of the film, and for the better, as Napper said."  @KKtheFilm #SMA #SpinalMuscularAtrophy #MuscularDystrophy #RareDisease #Evrysdi  KrisandKyletheFilm.com SnappyClothing.com Evrysdi.com Listen to the podcast here

Patricia Panzarino, otherwise known as Pidgie, was born with spinal muscular atrophy, SMA, a severe progressive neuromuscular disease. As a singer and songwriter and one of the oldest women living with SMA, Pidgie is celebrating the release of her new album, Just Breathe. Her experience of successfully taking Evrysdi from Genentech has given her more energy and enthusiasm. Pidgie elaborates, "They didn't know as much when I was born. I was six months old and did not achieve the milestone of crawling properly. My mom took me to the doctor. I had an older sister who had it as well. She was 12 years older than me, so they said, "Here we go again." They called it Amyotonia back then. But as far as a solid diagnosis of blood tests and DNA and stuff, truthfully, that was three years ago. But SMA used to be diagnosed clinically. So I knew I had it, but it was a different way to diagnose it." "Before the medication, you're talking 61 years of lifestyle—great parents kept me active. My father adapted things for me to participate more fully. For example, we had a boat that you could sleep on with a flying bridge, and he took a Hoyer lift that you use to transfer, put it on the flying bridge, and hoisted me up, so I could pilot this 31-foot boat. I was kept very active, taking vitamins and eating healthily. I never really did drugs or got really into alcohol. I just tried to keep a healthy balance and positivity until the medication. Now I'm still doing all of that because that really helps. You can't take it and just party and not eat well and stay up late and not sleep." #PidgieMusic #Pidgie #SpinalMuscularAtrophy #SMA #NeuromuscularDisease #Evrysdi PidgieMusic.com  Download the transcript here  

Patricia Panzarino, otherwise known as Pidgie, was born with spinal muscular atrophy, SMA, a severe progressive neuromuscular disease. As a singer and songwriter and one of the oldest women living with SMA, Pidgie is celebrating the release of her new album, Just Breathe. Her experience of successfully taking Evrysdi from Genentech has given her more energy and enthusiasm. Pidgie elaborates, "They didn't know as much when I was born. I was six months old and did not achieve the milestone of crawling properly. My mom took me to the doctor. I had an older sister who had it as well. She was 12 years older than me, so they said, "Here we go again." They called it Amyotonia back then. But as far as a solid diagnosis of blood tests and DNA and stuff, truthfully, that was three years ago. But SMA used to be diagnosed clinically. So I knew I had it, but it was a different way to diagnose it." "Before the medication, you're talking 61 years of lifestyle—great parents kept me active. My father adapted things for me to participate more fully. For example, we had a boat that you could sleep on with a flying bridge, and he took a Hoyer lift that you use to transfer, put it on the flying bridge, and hoisted me up, so I could pilot this 31-foot boat. I was kept very active, taking vitamins and eating healthily. I never really did drugs or got really into alcohol. I just tried to keep a healthy balance and positivity until the medication. Now I'm still doing all of that because that really helps. You can't take it and just party and not eat well and stay up late and not sleep." #PidgieMusic #Pidgie #SpinalMuscularAtrophy #SMA #NeuromuscularDisease #Evrysdi PidgieMusic.com  Listen to the podcast here  

In episode 123, host Kevin Schaefer talks with returning guests LaMondre Pough and Gabrielle Runyon, two Black Disabled advocates. In recognition of Black History Month, LaMondre and Gabrielle share their experiences growing up with SMA, the intersectionality of their identities, and the work they're doing now. ================================ To learn more about Evrysdi, please visit http://www.ApprovedForSMA.com ================================ To take part in our ongoing discussions regarding SMA, please visit http://www.smanewstoday.com/forums

In episode 122, host Kevin Schaefer talks with three of his coworkers from BioNews Inc., the parent company of SMA News Today. Brittany Foster is BioNews' HR Coordinator, and she lives with pulmonary hypertension. Brad Dell is the Director of Community Content, and he lives with cystic fibrosis. Matt Lafleur is the Culture Coordinator, and he lives with Friedreich's ataxia. In anticipation of Rare Disease Day 2023, these advocates share their experiences, pitfalls, and triumphs living with their respective conditions. ================================ Brittany's column: https://pulmonaryhypertensionnews.com/recharged-and-rewired-brittany-foster/ Brad's column: https://cysticfibrosisnewstoday.com/victorious-brad-dell/ Matt's column: https://friedreichsataxianews.com/little-victories-matthew-lafleur/ ================================ To learn more about Evrysdi, please visit http://www.ApprovedForSMA.com ================================ To take part in our ongoing discussions regarding SMA, please visit http://www.smanewstoday.com/forums

In episode 121, host Kevin Schaefer talks with Patricia Panzarino, who goes by her musician name, Pidgie. Pidgie is a singer and songwriter from Cape Cod, Massachusetts, and she has SMA Type 2. She discusses growing up in a pre-ADA era, building her music career, and starting Evrysdi. ================================ To learn more about Evrysdi, please visit http://www.ApprovedForSMA.com ================================ To take part in our ongoing discussions regarding SMA, please visit http://www.smanewstoday.com/forums

In episode 120, host Kevin Schaefer talks with Dom Evans (he/they), LGBTQ and disability activist and filmmaker. Dom discusses his background, growing up with SMA, and how he became an activist. He also talks about his longtime partner and their relationship. Note: This podcast includes thoughts about suicide. If you are struggling with thoughts of suicide, please contact the Suicide Prevention Lifeline in the U.S. (988, or 988lifeline.org, available 24/7), Samaritans in the U.K (samaritans.org), or Samaritans of Singapore in Singapore (sos.org.sg). ================================ To learn more about Evrysdi, please visit http://www.ApprovedForSMA.com ================================ To take part in our ongoing discussions regarding SMA, please visit http://www.smanewstoday.com/forums

In episode 119, host Kevin Schaefer talks with Chaz Hayden, a young adult author with SMA. Chaz's debut novel, The First Thing About You, tells the story of a 15-year-old boy with SMA named Harris who wants to reinvent himself. Kevin and Chaz discuss themes in the book, the personal experiences that influenced the story, and Chaz's advice for other writers. ================================ Chaz's website: https://www.chazhayden.com/ The First Thing About You: https://www.chazhayden.com/the-first-thing-about-you ================================ To learn more about Evrysdi, please visit www.ApprovedForSMA.com ================================ To take part in our ongoing discussions regarding SMA, please visit www.smanewstoday.com/forums

In episode 117, host Kevin Schaefer talks with Delphine Andrews and Kevin Davis from North Carolina. Delphine has SMA and she began dating Kevin in early 2020. They discuss their careers, daily lives, and the nuances of their relationship. ================================ To learn more about Evrysdi, please visit http://www.ApprovedForSMA.com ================================ To take part in our ongoing discussions regarding SMA, please visit http://www.smanewstoday.com/forums

In episode 118, host Kevin Schaefer talks with work colleagues Michael Morale and DeAnn Runge about aging and SMA. They start by discussing Scholar Rock's experimental therapy, Apitegromab, and go over recent updates surrounding its clinical trial. Then they talk about how SMA affects the way people think about getting older. Michael, DeAnn, and Kevin share their thoughts on aging as adults with SMA. ================================ Apitegromab news story: https://smanewstoday.com/news/apitegromab-potential-ease-fatigue-aid-life-quality-topaz/ Forum discussion on aging and SMA: https://smanewstoday.com/forums/forums/topic/thoughts-on-aging/ Kevin's column: https://smanewstoday.com/columns/how-sma-affected-perspective-aging-disability/ ================================ To learn more about Evrysdi, please visit http://www.ApprovedForSMA.com ================================ To take part in our ongoing discussions regarding SMA, please visit http://www.smanewstoday.com/forums

In episode 116, host Kevin Schaefer talks with Shaniqua Granby from Virginia Beach, Virginia. Shaniqua is an equal employment opportunity specialist, and she has SMA Type 3. She discusses going to law school, her faith and how it relates to her disability, and the advice she has for others with SMA. ================================ To learn more about Evrysdi, please visit http://www.ApprovedForSMA.com ================================ To take part in our ongoing discussions regarding SMA, please visit http://www.smanewstoday.com/forums

In episode 115, host Kevin Schaefer talks with Jose Flores from Miami, Florida. Jose is a bestselling author and motivational speaker who lives with SMA Type 3. He discusses his journey toward entrepreneurship, his marriage and family life, and becoming connected to the SMA community. ================================ Jose's website: https://www.joseinspires.com/ ================================ To learn more about Evrysdi, please visit http://www.ApprovedForSMA.com ================================ To take part in our ongoing discussions regarding SMA, please visit http://www.smanewstoday.com/forums

In episode 114, host Kevin Schaefer talks with Daniele Johnson from Evansville, Indiana. Daniele and her husband's youngest son, Anthony, has SMA type 0. She talks about her son's diagnosis, pursuing treatment, and how she and her family have adapted to life with SMA. ================================ To learn more about Evrysdi, please visit http://www.ApprovedForSMA.com ================================ To take part in our ongoing discussions regarding SMA, please visit http://www.smanewstoday.com/forums

In episode 113, host Kevin Schaefer talks with Dustin Swafford from Georgia. Dustin has been playing and coaching a power soccer team for years, and his team recently won the national championship. He discusses his love of sports, having his brother as his primary caregiver, and starting his own financial business. ================================ To learn more about Evrysdi, please visit http://www.ApprovedForSMA.com ================================ To take part in our ongoing discussions regarding SMA, please visit http://www.smanewstoday.com/forums

In episode 112, host Kevin Schaefer talks with Lexi Villa from Torrance, California. Lexi works in retail and shares her life with SMA on social media. She discusses moving away from home at a young age, becoming connected with the SMA community, and falling in love with her best friend from childhood. ================================ To learn more about Evrysdi, please visit http://www.ApprovedForSMA.com ================================ To take part in our ongoing discussions regarding SMA, please visit http://www.smanewstoday.com/forums

In episode 111, host Kevin Schaefer talks with Steven Verdile, a graphic designer and media creator from New York City. Steven is also the founder and editor-in-chief of The Squeaky Wheel, an online disability satire publication. He discusses living with SMA, working for NBC, and disability representation in media. ================================ Steven's website: https://www.stevenverdile.com/ Steven's IG: https://www.instagram.com/stevenverdilecreative/ The Squeaky Wheel: https://the-squeaky-wheel.com/ ================================ To learn more about Evrysdi, please visit http://www.ApprovedForSMA.com ================================ To take part in our ongoing discussions regarding SMA, please visit http://www.smanewstoday.com/forums

In episode 110, host Kevin Schaefer welcomes back to the podcast Ben Mattlin, an author and disability rights advocate. His upcoming book is called “Disability Pride: Dispatches from a Post-ADA World,” and is set to release on November 15, 2022. Ben discusses the evolution of this book, and what Disability Pride means to him. ================================ Pre-order Disability Pride: https://www.penguinrandomhouse.com/books/676271/disability-pride-by-ben-mattlin/ Ben's website: www.benmattlin.com ================================ To learn more about Evrysdi, please visit http://www.ApprovedForSMA.com ================================ To take part in our ongoing discussions regarding SMA, please visit http://www.smanewstoday.com/forums

In episode 109, host Kevin Schaefer chats to several attendees from this year's Cure SMA conference in Anaheim, California. Ashley Fox, Ryan Manriquez, and Kyle Harris discuss reuniting after such a long time, connecting with other adults with SMA, and share their highlights from the conference. ================================ To learn more about Evrysdi, please visit http://www.ApprovedForSMA.com ================================ To take part in our ongoing discussions regarding SMA, please visit http://www.smanewstoday.com/forums

In episode 108, host Kevin Schaefer talks with Logan Nantz from southern Indiana. Logan's daughter Hayden is two years old and has SMA. They discuss Logan family's story, as well as the news that Genentech recently approved Evrysdi for infants under the age of two months. ================================ Evrysdi story: https://smanewstoday.com/news-posts/2022/06/01/sma-treatment-evrysdi-gets-fda-approval-babies-under-2-months-old/ Logan's 31 Days of SMA story: https://smanewstoday.com/31-days-of-sma/2020/08/07/31-days-of-sma-logan-nantz/ ================================ To take part in our ongoing discussions regarding SMA, please visit www.smanewstoday.com/forums

In episode 107, host Kevin Schaefer talks about mental health with LGBTQ+ and disability advocates. Panelists include Ali Ramos (she/her), licensed social worker; Sherry Toh (she/they), columnist for SMA News Today; and Hugo Trevino (he/him), who works in academia. They all share their experiences managing their own mental health and offer advice for others in disability and LGBTQ+ spaces. ================================ Ali's Work Facebook page: https://www.facebook.com/aliramostherapy Sherry's column: https://smanewstoday.com/wandering-the-lines-sherry-toh/ Hugo's Instagram: https://www.instagram.com/htrevin2/ ================================ To learn more about Evrysdi, please visit http://www.ApprovedForSMA.com ================================ To take part in our ongoing discussions regarding SMA, please visit www.smanewstoday.com/forums ================================ For the most up-to-date information regarding SMA, please visit http://www.smanewstoday.com

In episode 106, host Kevin Schaefer talks with a group of rare disease and disability advocates about women's mental health. Panelists include Charlene Marshall, licensed mental health professional, and columnist for Pulmonary Fibrosis News; Brianna Albers, columnist for SMA News Today; Gabrielle Runyon, a college student with SMA at the University of Louisville; and Jessie Madrigal writer for endometriosis.net and mental health advocate. They all share their experiences managing their mental health and offer advice for others in disability spaces. ================================ Charlene's column: https://pulmonaryfibrosisnews.com/living-with-ipf-charlene-marshall/ Brianna's column: https://smanewstoday.com/the-wolf-finally-frees-itself-brianna-albers/ Gabrielle's Instagram: https://www.instagram.com/gab.davetta/ Jessie's Instagram: https://www.instagram.com/weemissjess ================================ To learn more about Evrysdi, please visit http://www.ApprovedForSMA.com ================================ To take part in our ongoing discussions regarding SMA, please visit www.smanewstoday.com/forums ================================ For the most up-to-date information regarding SMA, please visit www.smanewstoday.com

In episode 105, host Kevin Schaefer talks with rare disease and disability advocates about men's mental health. Panelists include Paris Dancy, licensed mental health professional, and columnist for Cushing's Disease News; Matt Lafleur, columnist for Friedreich's Ataxia News; Michael Morale, multimedia specialist for SMA News Today; and Ty Dykema, artist and disability advocate. They all share their experiences managing their mental health and offer advice for others in disability spaces. ================================ Paris' column: https://cushingsdiseasenews.com/surviving-and-thriving-paris-dancy/ Matt's column: https://friedreichsataxianews.com/little-victories-matthew-lafleur/ Michael's videos: https://www.youtube.com/playlist?list=PLtRCVbeiSodlFLsjQBls3fM_EOnm4o6GV Ty's Instagram pages: https://www.instagram.com/breachthelevee/ https://www.instagram.com/tmd.art/ ================================ To learn more about Evrysdi, please visit http://www.ApprovedForSMA.com ================================ To take part in our ongoing discussions regarding SMA, please visit http://www.smanewstoday.com/forums ================================ For the most up-to-date information regarding SMA, please visit http://www.smanewstoday.com

In episode 104, host Kevin Schaefer talks with Ben Lou from San Diego, California. Ben is a student at MIT, planning to double major in math and physics. He discusses his lifelong love for learning, growing up with SMA, and the importance of disability representation in STEM education. ================================ Article about Ben: https://smanewstoday.com/news-posts/2021/10/11/sma-ben-lou-pursuing-academic-dreams-mit/ ================================ To learn more about Evrysdi, please visit www.ApprovedForSMA.com ================================ To take part in our ongoing discussions regarding SMA, please visit www.smanewstoday.com/forums ================================ For the most up-to-date information regarding SMA, please visit www.smanewstoday.com

In episode 103, host Kevin Schaefer talks with Stephen Mikita and Arya Singh, two individuals with SMA who have years of experience with clinical trials. Stephen, 66, was an Assistant Attorney General for the state of Utah for more than 30 years, representing the three largest state agencies providing services and protections for individuals with disabilities. As one of the oldest survivors of SMA, Mr. Mikita has been uniquely positioned to advocate for the patient perspective at every stage of drug development. Arya is a senior at Yale and has dedicated much of her childhood to clinical research. Her parents are the founders of The SMA Foundation, which has brought some of the world's greatest scientists into SMA research. Arya herself has been a part of clinical research, and this experience was the inspiration behind a children's book, Courageous Calla & The Clinical Trial. She is a member of the New Haven Commission on Disabilities, with plans to obtain her undergrad degree this spring and a master's on public health next year. ================================ The SMA Foundation: https://smafoundation.org/ Arya's book: https://www.amazon.com/Courageous-Calla-Clinical-Trial-Singh/dp/B0892B4D8G ================================ To learn more about Evrysdi, please visit http://www.ApprovedForSMA.com ================================ To take part in our ongoing discussions regarding SMA, please visit http://www.smanewstoday.com/forums ================================ For the most up-to-date information regarding SMA, please visit www.smanewstoday.com

In this episode, host Kevin Schaefer talks to Amber-Joi Watkins, an SMA mom and advocate. Amber-Joi talks about raising her daughter, Celine, and becoming part of the SMA community. She also discusses her daughter's treatment journey, their daily lives, and shares some advice she has for other parents. ================================ To learn more about Evrysdi, please visit http://www.ApprovedForSMA.com ================================ To take part in our ongoing discussions regarding SMA, please visit http://www.smanewstoday.com/forums ================================ For the most up-to-date information regarding SMA, please visit http://www.smanewstoday.com

In this episode, host Kevin Schaefer talks with John Milligan from Austin, Texas. John is a Senior Manager in RND at Asuragen, a biotechnology company. They chat about the state of SMA testing, newborn screenings, the evolution of SMA research, and his day-to-day life. ================================ Learn more about Asuragen: https://asuragen.com/ ================================ To learn more about Evrysdi, please visit http://www.ApprovedForSMA.com ================================ To take part in our ongoing discussions regarding SMA, please visit http://www.smanewstoday.com/forums

SMA News Today's multimedia associate, Price Wooldridge, discusses how the FDA is reviewing a request that the use of Evrysdi, an oral and at-home SMA treatment, be extended to pre-symptomatic babies. Also, although exciting, getting a new wheelchair isn't always easy. DeAnn can relate to columnist Brianna Albers recent article, “A Year Later, I'm Still Wearing Old Shoes.” She goes into detail about the process and what she continues to struggle with. Are you interested in learning more about spinal muscular atrophy? If so, please visit https://smanewstoday.com/

In episode 100, host Kevin Schaefer talks with Gabrielle Runyon (she/her) from Louisville, Kentucky. Gabrielle is a sophomore at the University of Louisville, studying psychology. She talks about growing up with SMA, transitioning to college, and advocating for disabled individuals. ================================ Gabrielle's 31 Days of SMA Story: https://smanewstoday.com/31-days-of-sma/2020/08/24/31-days-of-sma-gabrielle-runyon/ ================================ To learn more about Evrysdi, please visit http://www.ApprovedForSMA.com ================================ To take part in our ongoing discussions regarding SMA, please visit http://www.smanewstoday.com/forums ================================ For the most up-to-date information regarding SMA, please visit http://www.smanewstoday.com

In episode 99, host Kevin Schaefer talks with two mothers from the SMA community. Trudy Citovic lives in Oregon, and her daughter Alina has SMA. As an advocate for newborn screenings, she talks about the role SMA has played in her experience of motherhood. Carli Hamilton is a social media influencer and mother who has SMA. She lives in Utah with her husband and daughter. She shares her thoughts on becoming a mother and the nuances of parenting with a disability. ================================ Learn more about newborn screenings here: https://smanewstoday.com/news-posts/2021/12/03/muscular-dystrophy-canada-novartis-maritime-newborn-sma-screening-program/ ================================ To learn more about Evrysdi, please visit http://www.ApprovedForSMA.com ================================ To take part in our ongoing discussions regarding SMA, please visit http://www.smanewstoday.com/forums ================================ For the most up-to-date information regarding SMA, please visit http://www.smanewstoday.com

In episode 98, host Kevin Schaefer talks with fellow SMA News Today contributors about hiring and managing caregivers. Listen as Kevin talks with DeAnn Runge and Michael Morale about their experiences with home healthcare, and how they find the right people. These panelists also discuss two recent columns from our main website. ================================ Join the conversation about caregivers here: https://smanewstoday.com/forums/forums/topic/starting-the-search-for-new-caregivers/ ================================ Columns: - ‘Don't Quit Before the Miracle Happens' by Sherry Toh - https://smanewstoday.com/columns/2022/01/05/instead-new-years-resolutions-im-making-playlist/ - Sharing Our Stories With the SMA Community's Next Generation by Halsey Blocher - https://smanewstoday.com/columns/2022/01/07/sharing-our-stories-with-next-generation-sma-community/ ================================ To learn more about Evrysdi, please visit http://www.ApprovedForSMA.com ================================ To take part in our ongoing discussions regarding SMA, please visit http://www.smanewstoday.com/forums ================================ For the most up-to-date information regarding SMA, please visit http://www.smanewstoday.com

SMA News Today's multimedia associate, Price Wooldridge, discusses how The National Institute for Health and Care Excellence has recommended that Evrysdi be provided at low or no cost to eligible SMA patients in England. Also, the process of modifying a wheelchair for specific needs is no easy task. DeAnn Runge shares how difficult it's been for her simply to receive comfortable arm rest pads. Even after replacing them, they're not what she hoped for. She's now contemplating what her next action should be. Are you interested in learning more about spinal muscular atrophy? If so, please visit https://smanewstoday.com/

In episode 97, host Kevin Schaefer talks with Ali Ramos (she/her) from Amarillo, Texas. Ali is a licensed social worker and activist, focusing on both the disability and LGBTQ communities. She talks with Kevin about living with SMA, pursuing independence, and her advocacy work. ================================ Ali's Instagram: https://www.instagram.com/kittylegs/ Ali's Facebook page: https://www.facebook.com/aliramostherapy/ ================================ To learn more about Evrysdi, please visit www.ApprovedForSMA.com ================================ To take part in our ongoing discussions regarding SMA, please visit http://www.smanewstoday.com/forums ================================ For the most up-to-date information regarding SMA, please visit http://www.smanewstoday.com

SMA News Today's multimedia associate, Price Wooldridge, discusses an article about how blood levels of neurofilaments and the results of a nerve-muscle test may be biomarkers for SMA onset/severity and treatment response. Also, Sherry Toh's SMA News Today article, “As an Adult With SMA, I Need Access to Evrysdi, Too,” pulls at your heartstrings. DeAnn can relate to those feelings as it reminds her of when she was trying to access Spinraza. Are you interested in learning more about spinal muscular atrophy? If so, please visit https://smanewstoday.com/

In this episode, host Kevin Schaefer talks with Halsey Blocher and Heather Dye from Fort Wayne, Indiana. Halsey is a columnist for SMA News Today, and her mother Heather is a caregiver and advocate. During this conversation, they talk about the nuances of living with SMA, transitioning to adulthood, and advice they have for others in the SMA community. ================================ Halsey's column: https://smanewstoday.com/from-where-i-sit-halsey-blocher/ Heather's 31 Days of SMA story: https://smanewstoday.com/31-days-of-sma/2020/08/14/31-days-of-sma-heather-halsey-dye/ ================================ To learn more about Evrysdi, please visit http://www.ApprovedForSMA.com ================================ To take part in our ongoing discussions regarding SMA, please visit http://www.smanewstoday.com/forums ================================ For the most up-to-date information regarding SMA, please visit http://www.smanewstoday.com

In episode 95, host Kevin Schaefer talks with the creators of the new song “Spaces.” Done in collaboration with Genentech, this production was created entirely by people from the SMA community. James Ian is a singer, songwriter, and actor from Los Angeles, California. With Spaces, he wanted to create a song specifically for the disability community. Dominick Evans is a trans, non-binary creator originally from the Midwest. He has a BFA in Film and is a filmmaker, writer, gamer, streamer, YouTuber, parent, speaker, and activist. Zack and Zarek Elizondo are brothers, YouTubers, podcasters, and graphic designers. You can learn more about their lives and work on their website, the Sweet Life of Zack and Zarek. ================================ Link to “Spaces” music video: https://www.youtube.com/watch?v=ZLyRpDn9bTo ================================ To learn more about Evrysdi, please visit http://www.ApprovedForSMA.com ================================ To take part in our ongoing discussions regarding SMA, please visit http://www.smanewstoday.com/forums ================================ For the most up-to-date information regarding SMA, please visit http://www.smanewstoday.com

SMA News Today's multimedia associate, Price Wooldridge, discusses how Roche and Genentech will initiate a Phase 2/3 clinical trial to evaluate the safety and efficacy of GYM329 in combination with Evrysdi in children with SMA. Also, after thinking this Thanksgiving would resemble some normalcy, Covid throws a wrench into DeAnn's family plans. She explains why their plans have changed and her feelings regarding it. Are you interested in learning more about spinal muscular atrophy? If so, please visit https://smanewstoday.com/

In episode 94, host Kevin Schaefer talks with Pamela K. Muhammad from Monroe, Louisiana. Pamela is the author of two books, and she is currently in the process of launching her own fashion accessory business. She talks about living independently with SMA, combatting disability stereotypes, and shares advice for others in the community. ================================ Pamela's poetry book: https://www.amazon.com/Pam-Allahs-Poetry-Heart-Collection-Inspires/dp/170241292X/ref=sr_1_1?dchild=1&qid=1635261256&refinements=p_27%3APamela+K.+Muhammad&s=books&sr=1-1&text=Pamela+K.+Muhammad Pamela's memoir: https://www.amazon.com/Loving-Skin-Im-Visions-Life/dp/B08HGTSZXT/ref=sr_1_2?dchild=1&qid=1635261337&refinements=p_27%3APamela+K.+Muhammad&s=books&sr=1-2&text=Pamela+K.+Muhammad ================================ To learn more about Evrysdi, please visit www.ApprovedForSMA.com ================================ To take part in our ongoing discussions regarding SMA, please visit www.smanewstoday.com/forums ================================ For the most up-to-date information regarding SMA, please visit www.smanewstoday.com

In episode 93, host Kevin Schaefer talks with Garrett Lerner from Los Angeles, California. Garrett is the co-creator and co-showrunner of the NBC drama series “Ordinary Joe,” and he is a father to a son with SMA type II. He talks with Kevin about the origins of the show and its disability representation, his family life, and the importance of seeing disabled characters on screen. ================================ Watch Ordinary Joe: https://www.nbc.com/ordinary-joe ================================ To learn more about Evrysdi, please visit http://www.ApprovedForSMA.com ================================ To take part in our ongoing discussions regarding SMA, please visit www.smanewstoday.com/forums ================================ For the most up-to-date information regarding SMA, please visit http://www.smanewstoday.com

In episode 92, host Kevin Schaefer talks with fellow SMA News Today contributors to discuss each other's work experiences. October is recognized in the United States as National Disability Employment Awareness Month (NDEAM). Listen as Kevin talks with DeAnn Runge, Michael Morale, and Alyssa Silva about the nuances of working with a disability, finding jobs, and maintaining disability benefits while working. ================================ Join the conversation about NDEAM here: https://smanewstoday.com/forums/forums/topic/ndeam-what-was-your-first-job/ ================================ To learn more about Evrysdi, please visit http://www.ApprovedForSMA.com ================================ To take part in our ongoing discussions regarding SMA, please visit http://www.smanewstoday.com/forums ================================ For the most up-to-date information regarding SMA, please visit http://www.smanewstoday.com

SMA News Today's multimedia associate, Price Wooldridge, reads an article on how pre-symptomatic infants with SMA have retained the ability to swallow after being treated with Evrysdi for at least one year in a clinical trial. Also, October is National Disability Employment Awareness Month (NDEAM.) As such it got DeAnn thinking about one of her first jobs and how she obtained it. Employment opportunities are out there and by thinking outside the box, seeking a little guidance if need be and pursuing your passions it might just be a bit easier finding suitable work. Are you interested in learning more about spinal muscular atrophy? If so, please visit https://smanewstoday.com/

In episode 91, host Kevin Schaefer talks with Blake Watson from Byram, Mississippi. Blake is a web designer and developer. He is currently a member of the frontend dev team at MRI Technologies, working on projects for NASA. He has SMA Type II and is passionate about helping disabled individuals find employment. ================================ Blake's website: https://blakewatson.com Blake's Twitter: @blakewatson ================================ To learn more about Evrysdi, please visit www.ApprovedForSMA.com ================================ To take part in our ongoing discussions regarding SMA, please visit www.smanewstoday.com/forums ================================ For the most up-to-date information regarding SMA, please visit www.smanewstoday.com

SMA News Today's multimedia associate, Price Wooldridge, discusses a trial planned of Spinraza at high dose in Spinal Muscular Atrophy (SMA) patients who have used Evrysdi. Read the news article: https://smanewstoday.com/news-posts/2021/09/17/high-dose-spinraza-trial-sma-patients-using-evrysdi/ DeAnn Runge doesn't shy away from personal topics especially when she feels others can relate or offer advice. One of the added challenges, when you have a disability, is dealing with your period. She talks about how she manages it, but also discusses why she's looking for alternative options. Are you interested in learning more about spinal muscular atrophy? If so, please visit https://smanewstoday.com/

In episode 90, host Kevin Schaefer talks with Judy Heumann, who's originally from Brooklyn, New York. A pioneer of the disability rights movement, she is one of the primary subjects of the 2020 Netflix documentary Crip Camp. In the United States, she served in the administrations of Presidents Bill Clinton and Barack Obama. In 2020, she published her memoir Being Heumann: An Unrepentant Memoir of a Disability Rights Activist. ================================ Judy's book: https://www.amazon.com/Being-Heumann-Unrepentant-Disability-Activist/dp/0807019291 Judy's Instagram: https://www.instagram.com/theheumannperspective/ ================================ To learn more about Evrysdi, please visit http://www.ApprovedForSMA.com ================================ To take part in our ongoing discussions regarding SMA, please visit http://www.smanewstoday.com/forums ================================ For the most up-to-date information regarding SMA, please visit http://www.smanewstoday.com

In episode 89, host Kevin Schaefer talks with Carole St-Laurent from Quebec, Rimouski. Carole is the author of multiple children's books, for which she writes under the name Rainbow Gal. She talks about her artistic career and past life as a musician, living with SMA, and her experiences with traveling and living independently. ================================ Carole's website: http://Rainbowgal.com ================================ To learn more about Evrysdi, please visit http://www.ApprovedForSMA.com ================================ To take part in our ongoing discussions regarding SMA, please visit http://www.smanewstoday.com/forums ================================ For the most up-to-date information regarding SMA, please visit http://www.smanewstoday.com

Dan Temple is a patient advocate for those with Spinal Muscular Atrophy (SMA) and he shares his treatment journey and actions he has taken to raise awareness about SMA.  We also talk about the drug treatments that have come onto the scene since he was first diagnosed when Dan was 2 years old.  He is now 49. Dan says, "Like I said, very important was that my lung function improved. I probably gained about ten years back from what I lost. I'm still on a feeding tube, but I couldn't eat by mouth for seven years, and the drugs helped my muscles start working better. So I can eat and drink by mouth a little bit. I don't do it a lot, but it's nice to be able to do it occasionally and have some food that I enjoy. And my hand, of course. The one finger I have is kept pretty strong. Thank God because if I lose that, I've got nothing." "In general, it's kept me from getting worse, and when the drugs came along, I was at the point health-wise where I probably didn't have a lot of time left. My lungs were getting so weak that I was going to have to be on a respirator soon. So, basically, the Spinraza and Evrysdi both saved my life and gave me another 20 years, probably." #SMA #SpinalMuscularAtrophy #RareDisease #Evrysdi #Spinraza #MDA #MuscularDystrophyAssociation #PatientAdvocate #Genentech #Biogen #SMAAwarenessMonth  

Dan Temple is a patient advocate for those with Spinal Muscular Atrophy (SMA) and he shares his treatment journey and actions he has taken to raise awareness about SMA.  We also talk about the drug treatments that have come onto the scene since he was first diagnosed when Dan was 2 years old.  He is now 49. Dan says, "Like I said, very important was that my lung function improved. I probably gained about ten years back from what I lost. I'm still on a feeding tube, but I couldn't eat by mouth for seven years, and the drugs helped my muscles start working better. So I can eat and drink by mouth a little bit. I don't do it a lot, but it's nice to be able to do it occasionally and have some food that I enjoy. And my hand, of course. The one finger I have is kept pretty strong. Thank God because if I lose that, I've got nothing." "In general, it's kept me from getting worse, and when the drugs came along, I was at the point health-wise where I probably didn't have a lot of time left. My lungs were getting so weak that I was going to have to be on a respirator soon. So, basically, the Spinraza and Evrysdi both saved my life and gave me another 20 years, probably." #SMA #SpinalMuscularAtrophy #RareDisease #Evrysdi #Spinraza #MDA #MuscularDystrophyAssociation #PatientAdvocate #Genentech #Biogen #SMAAwarenessMonth

In episode 88, host Kevin Schaefer talks with fellow SMA News Today contributors Michael Morale and Katie Napiwocki. They discuss this year's 31 Days of SMA campaign, as well as other topics and news stories related to SMA Awareness Month. Finally, they highlight some recent columns from our main website. ================================ Links to 31 Days of SMA campaign and news stories: - 31 Days of SMA stories: https://smanewstoday.com/31-days-of-sma/ - MDA Launches New Podcast Hosted by SMA Advocate: https://smanewstoday.com/news-posts/2021/08/10/mda-new-podcast-host-sma-advocate-mindy-henderson/ - Advocates Lobby US Congress During Virtual Rare Disease Week: https://smanewstoday.com/news-posts/2021/08/03/advocates-lobby-us-congress-during-virtual-rare-disease-week-2021/ ================================ Links to columns: - ”Inviting People to Sit at the Table of My Disabled Life” by Katie Napiwocki: https://smanewstoday.com/columns/2021/07/22/relationships-inviting-people-sit-table-disabled-life/ - ”A Plea for Singapore to Approve SMA Treatments” by Sherry Toh: https://smanewstoday.com/columns/2021/08/04/plea-singapore-approve-sma-treatments/ - ”SMA Pride and Disability Language” by Kevin Schaefer: https://smanewstoday.com/columns/2021/08/10/sma-pride-disability-language/ ================================ To learn more about Evrysdi, please visit http://www.ApprovedForSMA.com ================================ To take part in our ongoing discussions regarding SMA, please visit http://www.smanewstoday.com/forums ================================ For the most up-to-date information regarding SMA, please visit http://www.smanewstoday.com

SMA News Today's multimedia associate, Price Wooldridge, discusses how the Zolgensma-Evrysdi combination is likely beneficial for patients with Spinal Muscular Atrophy (SMA) Type 1. Also, DeAnn Runge shares about the stress and anxiety that come along with verifying disability eligibility. Are you interested in learning more about spinal muscular atrophy? If so, please visit https://smanewstoday.com/

In episode 87, host Kevin Schaefer talks with Allie Williams from Stillwater, Oklahoma. Allie is a vocal coach and she has SMA Type II. She talks about her lifelong musical journey, dealing with accessibility issues as a college student, and advice she has for others with SMA. ================================ To learn more about Evrysdi, please visit http://www.ApprovedForSMA.com ================================ To take part in our ongoing discussions regarding SMA, please visit http://www.smanewstoday.com/forums ================================ For the most up-to-date information regarding SMA, please visit www.smanewstoday.com

In episode 86, host Kevin Schaefer talks with Price Wooldridge from Fort Worth, Texas. Price is a voiceover contributor for the SMA News Today flash briefings, and he lives with a rare autoimmune disease called Lambert-Eaton Myasthenic Syndrome. Price discusses his background and diagnosis story, the commonalities and differences between SMA and LEMS, and his advice for others living with rare diseases. ================================ To learn more about Evrysdi, please visit www.ApprovedForSMA.com ================================ To take part in our ongoing discussions regarding SMA, please visit www.smanewstoday.com/forums ================================ For the most up-to-date information regarding SMA, please visit www.smanewstoday.com

In episode 85, host Kevin Schaefer talks with Mikey and Noelle Hazel from Union, New Jersey. Mikey and Noelle are siblings who both have SMA Type II. They talk about growing up with SMA together, combating ableism, and entering a new chapter in their lives. ================================ Link to Mikey's YouTube Channel: https://www.youtube.com/channel/UCNx-ePIR4bO2tPKCSfPb9fQ ================================ To learn more about Evrysdi, please visit http://www.ApprovedForSMA.com ================================ To take part in our ongoing discussions regarding SMA, please visit http://www.smanewstoday.com/forums ================================ For the most up-to-date information regarding SMA, please visit http://www.smanewstoday.com

SMA News Today's multimedia associate, Price Wooldridge, discusses Japan's approval of Evrysdi as the first at-home, oral Spinal Muscular Atrophy (SMA) treatment. Also, DeAnn Runge talks about her quest to start pool therapy and the challenges she's faced with. It's just not as easy as dipping your toe in the water. Are you interested in learning more about spinal muscular atrophy? If so, please visit https://smanewstoday.com/

SMA News Today's multimedia associate, Price Wooldridge, discusses Evrysdi being linked to motor function improvements in a broad range of people with Spinal Muscular Atrophy (SMA). Also, Forums Director Kevin Schaefer reads a column about finding community support through this year's Cure SMA virtual conference. Link to Kevin's column: https://smanewstoday.com/category/embracing-my-inner-alien-a-column-by-kevin-schaefer/ Are you interested in learning more about spinal muscular atrophy? If so, please visit https://smanewstoday.com/

In episode 80, host Kevin Schaefer talks with fellow SMA News Today contributors Michael Morale and DeAnn Runge. They discuss some topics from the 2021 virtual Cure SMA conference and general news updates. The team also talks about restrictions being lifted across the U.S. and the impact of this on the SMA community. Finally, they highlight some recent columns from our main website. ================================ Links to news stories and forum topics: - Apitegromab Put on FDA's Fast Track https://smanewstoday.com/news-posts/2021/05/26/muscle-directed-therapy-apitegromab-put-on-fda-fast-track/ - SMA Added to North Carolina's Newborn Screening Program https://smanewstoday.com/news-posts/2021/06/09/sma-added-north-carolina-newborn-screening-program/ - Discussion on the film "Sound of Metal" https://smanewstoday.com/forums/forums/topic/have-you-watched-sound-of-metal/ ================================ Links to columns: - ”Embracing the Growing Pains of New Wheels” by Katie Napiwocki https://smanewstoday.com/columns/2021/06/03/new-power-wheelchair-embracing-growing-pains/ - ”I'm Taking My Time Returning to Normal” by Brianna Albers https://smanewstoday.com/columns/2021/06/07/taking-my-time-returning-normal/ - ”I Don't Want a World Without SMA. Here's Why.'” by Sherry Toh https://smanewstoday.com/columns/2021/06/09/gene-editing-world-without-sma/ ================================ To learn more about Evrysdi, please visit http://www.ApprovedForSMA.com ================================ To take part in our ongoing discussions regarding SMA, please visit http://www.smanewstoday.com/forums ================================ For the most up-to-date information regarding SMA, please visit http://www.smanewstoday.com

SMA News Today's multimedia associate, Price Wooldridge, discusses England's NICE recommending against adding Evrysdi to the UK's Public Health System. Also, DeAnn Runge reads Katie Napiwocki's recent column, “Embracing the Growing Pains of New Wheels.” From the hesitancy to start the process to finding ways to adjust once the transition happens DeAnn can relate to it all. Read Katie's column here: https://smanewstoday.com/columns/2021/06/03/new-power-wheelchair-embracing-growing-pains/ Are you interested in learning more about spinal muscular atrophy? If so, please visit https://smanewstoday.com/

SMA News Today's multimedia associate, Price Wooldridge, reads the column by Halsey Blocher, From Where I Sit, “SMA and Sibling Relationships: My Brother's Perspective”. Also, DeAnn Runge talks about how she came to the decision to stick with Evrysdi. It was a difficult decision but she's happy with her choice. Are you interested in learning more about spinal muscular atrophy? If so, please visit https://smanewstoday.com/

SMA News Today's multimedia associate, Price Wooldridge, discusses Apitegromab being granted a fast track designation by the U.S. Food and Drug Administration. DeAnn Runge talks about her upcoming appointment with her neurologist to discuss continuing with Evrysdi or switching back to Spinraza. She dreads making the wrong decision. Are you interested in learning more about spinal muscular atrophy? If so, please visit https://smanewstoday.com/

In episode 83, host Kevin Schaefer talks with Christine Getman and Scottie Foertmeyer from Portland, Oregon. Christine and Scottie are partners in life and in business, as they run the nonprofit Magic Wheelchair together. They talk about their relationship, advocacy work, and daily living. ================================ Link to Magic Wheelchair: https://www.magicwheelchair.org/ ================================ To learn more about Evrysdi, please visit www.ApprovedForSMA.com ================================ To take part in our ongoing discussions regarding SMA, please visit www.smanewstoday.com/forums ================================ For the most up-to-date information regarding SMA, please visit www.smanewstoday.com

We discuss the pathophysiology, presentation, and available and emerging treatment for patients with spinal muscular atrophy. We are joined by Diana Castro, MD, Associate Professor of Pediatrics and Neurology at UT Southwestern and Children’s Health Dallas.

In episode 82, host Kevin Schaefer talks with Hawken Miller from Newport Beach, California. Hawken is a features writer for SMA News Today’s parent company, BioNews, as well as a columnist for Muscular Dystrophy News Today. He discusses growing up with Duchenne MD, the evolution of his journalism career, and learning about the SMA community. ================================ Link to Hawken’s column: https://musculardystrophynews.com/category/hawks-eye-view-a-column-by-hawken-miller/ ================================ To learn more about Evrysdi, please visit http://www.ApprovedForSMA.com ================================ To take part in our ongoing discussions regarding SMA, please visit http://www.smanewstoday.com/forums ================================ For the most up-to-date information regarding SMA, please visit http://www.smanewstoday.com

In episode 81, host Kevin Schaefer talks with Chaz Hayden from Princeton, New Jersey. Chaz is a writer and YouTuber with SMA. He talks about growing up with SMA, starting college at a young age, and his upcoming young adult novel. ================================ Link to Chaz’s YouTube channel: https://www.youtube.com/channel/UCwPeqLbJO9s2v9UDrdbzbnQ ================================ Music Credits: Alec's Band A Young Griffin Boyle In Drag ================================ To learn more about Evrysdi, please visit https://www.ApprovedForSMA.com ================================ To take part in our ongoing discussions regarding SMA, please visit https://www.smanewstoday.com/forums ================================ For the most up-to-date information regarding SMA, please visit https://www.smanewstoday.com

In this week’s episode, we will be discussing the treatment journey in spinal muscular atrophy, or SMA, and the experience of taking SPINRAZA and Evrysdi, with Nick Sinagra, who is a patient himself with SMA. If you would like to nominate a pharmacist or pharmacy staff member as a Pharmacy Hero, email AAntrim@pharmacytimes.com!

SMA News Today’s multimedia associate, Price Wooldridge, discusses Health Canada’s approval of Evrysdi for at-home treatment in SMA patients. Also, Forums Director Kevin Schaefer reads a column by Ari Anderson about his friendship with a North Carolina senator. Link to Ari’s column: https://smanewstoday.com/columns/2021/04/22/importance-people-recognize-my-worth-senator-thom-tillis/ Are you interested in learning more about spinal muscular atrophy? If so, please visit https://smanewstoday.com/

PTC Therapeutics, through its partnership with the SMA Foundation and Genentech, won approval last year for Evrysdi, the first oral therapy for the rare neurodegenerative condition spinal muscular atrophy. Now, the SMA Foundation is working with the company to discover and develop regenerative treatments that can reverse the damage done by the disease. At the same time, the company is advancing its effort on the gene therapy front following its 2018 acquisition of Agilis Biotherapeutics. We spoke to Matthew Klein, chief development officer for PTC Therapeutics, about the company's work in SMA, its move into regenerative medicine, and its efforts to win approval in Europe and the United States for its first gene therapy.

In episode 80, host Kevin Schaefer talks with fellow SMA News Today contributors Michael Morale and DeAnn Runge. They discuss updates on Scholar Rock’s treatment, Apitegromab, as well as other news stories and forum topics. Finally, they highlight some recent columns from our main website. ================================ Links to news stories and forum topics: -Cost of Rare Diseases in US? Nearly $1 Trillion in 2019, EveryLife Foundation Finds: https://smanewstoday.com/news-posts/2021/03/30/rare-diseases-cost-us-nearly-1-trillion-economic-burden-2019-everylife-foundation-study/ - Apitegromab Over 1 Year Bolsters Motor Abilities of Types 2 and 3: TOPAZ Trial: https://smanewstoday.com/news-posts/2021/04/09/top-line-topaz-trial-data-support-apitegromab-muscle-targeted-scholar-rock-therapy/ - Sleep solutions: https://smanewstoday.com/forums/forums/topic/sleep-solutions-2/ - Trying to keep expectations in check: https://smanewstoday.com/forums/forums/topic/trying-to-keep-expectations-in-check/ ================================ Links to columns: -”Why I Never Had a Spinal Fusion: A Retrospective, 10 Years Later” by Sherry Toh: https://smanewstoday.com/columns/2021/04/14/spinal-fusion-retrospective-10-years-later/ -”An SMA Patient and a Pulmonologist Comment on the COVID-19 Vaccine” by Halsey Blocher: https://smanewstoday.com/columns/2021/03/26/covid-19-vaccine-comments-sma-patient-pulmonologist/ -”How to Build and Maintain a Network of ‘Masked Advocates’” by Ari Anderson: https://smanewstoday.com/columns/2021/03/25/building-maintain-network-masked-advocates/ ================================ Music Credits: Alec's Band A Young Griffin Boyle In Drag ================================ To learn more about Evrysdi, please visit http://www.ApprovedForSMA.com ================================ To take part in our ongoing discussions regarding SMA, please visit http://www.smanewstoday.com/forums ================================ For the most up-to-date information regarding SMA, please visit http://www.smanewstoday.com

Dr. Dalia Moawad, MD, Executive Director & Disease Area Lead for Neurological Rare Diseases at Genentech discusses the three things her team learned during the pandemic from the launching of Evrysdi (risdiplam) the first at-home oral medicine for adults and children two months of age and older with spinal muscular atrophy (SMA). Dalia Moawad, M.D. Executive Director, Head of Neurological Rare Diseases, Medical Affairs. Dalia oversees the neurological rare disease portfolio for Genentech. At Genentech she has lead a portfolio of 10 established products, including most recently for the Evrysdi and the launch of the first influenza antiviral to come to market in 18 years. Between 2011 and 2014, she held several positions in early development and global clinical research with Bristol Myers Squibb and FivePrime Therapeutics. She spent 3 years at UCSF Gastroenterology Division focused on hepatology research before joining Genentech in 2006 with the anti-CD20 programs in immunology, first in late stage clinical development then medical affairs. Dalia holds an MBBCh from School of Medicine, Ain Shams University in Cairo. #Neuroscience #Evrysdi

SMA News Today’s multimedia associate, Price Wooldridge, discusses Evrysdi’s approval in Europe as the first oral, at-home treatment for Spinal Muscular Atrophy (SMA). Also, DeAnn Runge reads the latest column from Brianna Albers as she continues the quest to start the SMA treatment Evrysdi, The Evrysdi Chronicles: Full Speed Ahead. Are you interested in learning more about spinal muscular atrophy? If so, please visit https://smanewstoday.com/

In episode 79, host Kevin Schaefer talks with Ainaa Farhanah from Kuala Lumpur, Malaysia. Ainaa is a graphic designer and art teacher with SMA. She talks about growing up with SMA, family, college, and advocating for others with disabilities. Note: Ainaa does have limited speech capabilities, but we chose to leave the recording as it is. We at SMA News Today want to provide our listeners with a range of voices. It is our responsibility to highlight the diversity within the SMA community. You can find the transcript of this conversation here: http://bit.ly/SMA-Podcast79-Transcript ================================ Link to Ainaa’s Instagram: https://www.instagram.com/ainaafrhanah/tagged/ ================================ Music Credits: Alec's Band A Young Griffin Boyle In Drag ================================ To learn more about Evrysdi, please visit http://www.ApprovedForSMA.com ================================ To take part in our ongoing discussions regarding SMA, please visit http://www.smanewstoday.com/forums ================================ For the most up-to-date information regarding SMA, please visit http://www.smanewstoday.com

WBZ's Jordan Rich shares some good news for Spinal Muscular Atrophy sufferers.

Here are the links for everything discussed in Episode 41, I'm also including times here so feel free to skip ahead to the topics that interest you. (1:18) Monjuvi approved for DLBCL (3:07) Approval of Lampit to treat Chagas disease (4:52) Evrysdi approved for spinal muscular atrophy (8:13) FDA approved Blenrep for multiple myeloma (10:09) Approval of Viltepso for Duchenne Muscular Dystrophy CDC updates on COVID-19 & influenza reportingConnect with The Rx Daily Dose:Twitter      Instagram      YouTube      Linkedin       WebsiteEmail: therxdailydose@gmail.comConnect with Ian Parnigoni PharmD. on social media:Twitter       Instagram       Linkedin  ★ Support this podcast on Patreon ★

People with the rare and fatal genetic disease spinal muscular atrophy in recent years have seen the approval of an antisense therapy as well as a gene therapy. Genentech has now won U.S. Food and Drug Administration approval for Evrysdi, the first oral, at-home treatment for the condition. We spoke to Levi Garraway, chief medical officer and head of global product development at Genentech, about Evrysdi, how it works, and how it fits into the choices physicians and patients have when it comes to treating spinal muscular atrophy.