Podcasts about AveXis

Dr. Stephen Moran, Ph.D., is Global Program Head, Neuroendocrine Tumors & Other Radiosensitive Cancers, for Advanced Accelerator Applications (AAA - https://www.adacap.com/), a Novartis company and also a member of the Oncology Development Unit Leadership Team at Novartis. Prior to joining AAA, Dr. Moran was Global Head of Novartis Strategy, where he played a key role in defining the company's strategy, prioritizing critical actions needed to deliver on the mission to discover new ways to extend and improve peoples' lives. He also led numerous strategic initiatives, including gene therapy (AveXis, now Novartis Gene Therapies), RNA therapeutics (The Medicines Company), precision medicine and digital strategies. Dr. Moran joined Novartis as Strategic Assistant to the CEO, a position he held for two years and prior to this, he was an associate principal at McKinsey & Company serving as a leader in the healthcare practice, where he focused on health system sustainability, research and development strategy, and the economic analysis of clinical interventions across disease pathways. Dr. Moran holds a Bachelor of Arts and a Master of Science in Biochemistry from the University of Cambridge in the United Kingdom, including an undergraduate exchange program at the Massachusetts Institute of Technology (MIT). He also received a Doctorate from the University of Oxford in Biophysics where he lectured on thermodynamics, quantum mechanics and electromagnetism as applied to biology. Dr. Moran has also been a guest lecturer in strategy at the University of St. Gallen International Executives MBA, masters and bachelor courses, and received the Young Investigator of the Year award in 2006 from Biochemical Journal.

Picture this. A pandemic. You and your girlfriend of 2 years are forced closer than ever before. Ava Capri Palazzolo shares how she and her girlfriend managed to be one of the few couples to make it through this panni. We then travel back to the beginning, and answer some questions about the time their relationship was a secret, and how the public and painful breakups of popular lesbian couples influenced their decision to stay private. We then do a deeper dive into how YouTube can impact a relationship, and why Avexis will never have a joint channel (unless they're really strapped for cash).  Thank you to our sponsors! Curology: curology.com/dating to unlock a free 30-day trial Better Help: betterhelp.com/dating for 10% off your first month Best Fiends: Bestfiends.com to download the game for free  Aura Frames: auraframes.com code DATING for up to $100 off now until Mother's Day.   See omnystudio.com/listener for privacy information.

Picture this. A pandemic. You and your girlfriend of 2 years are forced closer than ever before. Ava Capri Palazzolo shares how she and her girlfriend managed to be one of the few couples to make it through this panni. We then travel back to the beginning, and answer some questions about the time their relationship was a secret, and how the public and painful breakups of popular lesbian couples influenced their decision to stay private. We then do a deeper dive into how YouTube can impact a relationship, and why Avexis will never have a joint channel (unless they’re really strapped for cash).  Thank you to our sponsors! Curology: curology.com/dating to unlock a free 30-day trial Better Help: betterhelp.com/dating for 10% off your first month Best Fiends: Bestfiends.com to download the game for free  Aura Frames: auraframes.com code DATING for up to $100 off now until Mother's Day.   See omnystudio.com/listener for privacy information.

Nancy L. Kuntz, MD, FAAN, Professor of Pediatrics and Neurology at Northwestern University Feinberg School of Medicine provides an overview of best practices to treat spinal muscular atrophy (SMA). This CME activity is possible through an educational grant from AveXis. To obtain credit for this activity, please visit https://checkrare.com/learning/p-spinal-muscular-atrophy-module2-managing-sma/

Nancy L. Kuntz, MD, FAAN, Professor of Pediatrics and Neurology at Northwestern University Feinberg School of Medicine provides an overview of the importance of genetic counseling for spinal muscular atrophy (SMA). This CME activity is possible through an educational grant from AveXis. To obtain credit for this activity, please visit https://checkrare.com/learning/p-spinal-muscular-atrophy-module4-genetic-counseling-sma/

Nancy L. Kuntz, MD, FAAN, Professor of Pediatrics and Neurology at Northwestern University Feinberg School of Medicine provides an overview of best practices to diagnose spinal muscular atrophy (SMA). This CME activity is possible through an educational grant from AveXis. To obtain credit for this activity, please visit https://checkrare.com/learning/p-spinal-muscular-atrophy-module1-diagnosing-sma/

Nancy L. Kuntz, MD, FAAN, Professor of Pediatrics and Neurology at Northwestern University Feinberg School of Medicine provides an overview of the importance of newborn screening for spinal muscular atrophy (SMA). This CME activity is possible through an educational grant from AveXis. To obtain credit for this activity, please visit https://checkrare.com/learning/p-spinal-muscular-atrophy-module3-newborn-screening-sma/

The economics of developing gene therapies can make it unattractive for biopharmaceutical companies to invest in bringing a gene therapy through development and to the market for ultra-rare conditions. But researchers at the National Center for Advancing Translational Sciences are working to develop a set of gene therapy vectors that can be used in multiple indications and eliminate the time and cost of preclinical development for a range of conditions through its Platform Vector Gene Therapy, or PaVe-GT program. In this fourth and final part of our gene therapy series, we spoke to P.J. Brooks, program director in the Office of Rare Diseases Research at the National Center for Advancing Translational Sciences, about the PaVe-GT program, the potential for developing a toolkit of plug-and-play vectors, and how this can alter the cost of developing gene therapies for ultra-rare and individual patients. This series is made possible through support from BioMarin, Pfizer, Retrophin, Novartis Gene Therapies (formerly AveXis), UCB Inc., Genentech, Ultragenyx, Novartis, RegenxBIO, and Sangamo Therapeutics.

Though there are only a handful of gene therapies on the market today, there is a robust and growing pipeline of these transformative medicines advancing toward market. In this third part of our gene therapy series we spoke to Janet Lambert, CEO of the Alliance for Regenerative Medicine, about the state of the gene therapy industry, the challenges developers face in advancing therapies to the market, and emerging pricing approaches to make them accessible to the patients who need them. This series is made possible through support from BioMarin, Pfizer, Retrophin, Novartis Gene Therapies (formerly AveXis), UCB Inc., Genentech, Ultragenyx, Novartis, RegenxBIO, and Sangamo Therapeutics.

Genetic counselors play a unique role in the medical life of a person with a rare disease. They can serve as guide, translator, and trusted advisor. In this first part of our four part series on gene therapies, we spoke to genetic counselor Stephanie Gandomi, a Global Genes advocacy resource, about gene therapies, the role genetic counselors play in working with rare disease patients, and the considerations rare disease patients may make about whether to pursue a gene therapy through a clinical trial. This series is made possible through support from BioMarin, Pfizer, Retrophin, Novartis Gene Therapies (formerly AveXis), UCB Inc., Genentech, Ultragenyx, Novartis, RegenxBIO, and Sangamo Therapeutics.

SMA News Today’s multimedia associate, Price Wooldridge, discusses AveXis, now known as Novartis Gene Therapies, focusing on continued work with genetic diseases. Are you interested in learning more about spinal muscular atrophy? If so, please visit https://smanewstoday.com/

This episode discusses patient access to AveXis' gene therapy Zolgensma from Orsini Healthcare, US based specialty pharmacy's perspective. The senior team at Orsini provide insight on their experience of Zolgensma access including key manufacturer criteria, payer landscape along with challenges and lessons learnt. Presenter: Aparna Krishnan Contributors: Dave Frobel, Senior Vice President, Trade & Tom Shaughnessy, Senior Vice President, Health Plans Producer: Aparna Krishnan About Orsini Healthcare: Founded in 2009, Orsini is a leading independent specialty pharmacy focused on rare conditions and gene therapies. Orsini was one of the first specialty pharmacies to provide Zolgensma, the first gene therapy for Spinal Muscular Atrophy. Today, Orsini has access to 40 limited distribution drugs, has more than 525 direct payor contracts and is recognized as a reimbursement expert in managing both medical and pharmacy benefits.

Sally Dyer is Vice President of Manufacturing Operations for the AveXis Longmont Manufacturing Center. Avexis is dedicated to developing and commercializing gene therapies for patients and families devastated by rare and life-threatening neurological genetic diseases. Sally was appointed to this role in April of 2019. She is accountable for the overall business and performance of the site. She works with a highly skilled team of leaders who use the company values of Integrity, Tenacity and Teamwork to delivery high quality products to our patients. On this week's show Sally shares her own road into her current role, the important work AveXis is doing, the current moment of uncertainty and how her company and industry plays its role, and much more. Hosted by Colorado Business Roundtable President Debbie Brown. 

The guys discuss Wind Chimes and who owns them. Apparently Eric owns three, though he's not sure he knows. In the Buzz around town, they discuss the Union Pointe Apartments, closure of Bin 46, AveXis, and Poppy Salon.In the Deep Dive, the guys get up close with recycling and reuse. If you need to get rid of something, check out some great resources from the City of Longmont, Eco-Cycle and Resource Central. Waste Diversion ResourcesCity of Longmont Waste DiversionEco-Cycle CHaRM (Center for Hard to Recycle Materials)Resource CentralHousehold Hazardous WasteEco-Cycle Resource GuideBusiness mentions In this episodeEco-CycleResource CentralWestern DisposalPeaceful Poppy SalonUnion Pointe ApartmentsAveXisEventsHandPan - check out this video to get an idea of Hand Pan Playing. Someone is providing instructions on this but the guys don't remember the name or contact. Check out Luna Cafe for a flyer on the subjectAdult Spelling Bee at 300 SunsMusicThanks to Andy Eppler for "Nothing but the Rain" as our IntroMusic of the week by Clandestine Amigo. Give a listen here to more of their music Check out Andy at his Instagram feed @andotheartist or his etsy shop

We discuss how AveXis is unaware of the cause of inflammation, that led to the STRONG trial hold of AVXS-101. Forums Director Kevin Schaefer shares some of his highlights from this year. Are you interested in learning more about the latest treatment for spinal muscular atrophy? Visit TreatSMA.com to see how this treatment works, hear about family stories, and learn about the steps to starting treatment. Visit TreatSMA.com.

In episode 46, hosts Kevin Schaefer and Michael Morale discuss the latest news regarding Roche and Genentech’s experimental therapy Risdiplam. We also talk about AveXis’ decision to put its STRONG trial on temporary hold, and some of our recent columns.

Dr. Meredith Schultz with AveXis discusses the FDA approval of Zolgensma (onasemnogene abeparvovec-xioi), the first gene therapy for the treatment of spinal muscular atrophy (SMA) in pediatric patients less than 2 yrs of age. It is given as a one-time infusion into the vein. Zolgensma was not evaluated in patients with advanced SMA.

We discuss how additional space for Zolgensma production is the goal of AveXis Catalent partnership and Forums Director Kevin Schaefer talks about some of the trending topics in the SMA News Today forums. Are you interested in learning more about the latest treatment for spinal muscular atrophy? Visit TreatSMA.com to see how this treatment works, hear about family stories, and learn about the steps to starting treatment. Visit TreatSMA.com.

We discuss how AveXis’ OneGene program could smooth the path for families wanting Zolgensma and Community Development Manager Kevin Schaefer talks about some trending discussions in the SMA News Today forums.

Hosts Kevin Schaefer and Michael Morale discuss a recent webinar about AveXis’ gene therapy Zolgensma. Then in part two, they talk about SMA awareness month, and a couple of our recent columns.

The FDA is investigating Novartis' subsidiary AveXis because AveXis may have manipulated data on Zolgensma, a gene therapy with a sky-high price tag and blockbuster potential. Here's what could happen next. Also, Exact Sciences goes all in on cancer diagnostics. Why the Cologuard-maker is spending $2.7 billion in a push deeper into genetic testing. Check out more of our content here: TMF's podcast portal YouTube Twitter Join Our Motley Fool Podcast Facebook Group LinkedIn StockUp, The Motley Fool's weekly email newsletter

We discuss an interview with AveXis where they state that Zolgensma is showing strong efficacy across SMA types. Also, Community Development Manager Kevin Schaefer talks about coping with bad days when you have SMA. Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful information about gene therapy, including its history and how it is being investigated for the treatment of genetic diseases. Visit www.exploregenetherapy.com

In this week’s episode, John shares some of the results from our fifth annual ACO Executive Survey. Results discussed include: ACO priorities 2019 initiatives Operational issues Factors affecting quality and cost Electronic health record interoperability Partnering activity About Darwin Research Group Darwin Research Group Inc. provides advanced market intelligence and in-depth customer insights to health care executives, with a strategic focus on health care delivery systems and the global shift toward value-based care. Darwin’s client list includes forward-thinking biopharmaceutical and medical device companies, as well as health care providers, private equity, and venture capital firms. The company was founded in 2010 as Darwin Advisory Partners, LLC and is headquartered in Scottsdale, Ariz. with a satellite office in Princeton, N.J.

In episode 36 of the SMA News Today Podcast, hosts Kevin Schaefer and Michael Morale discuss the FDA-approval of AveXis’ drug Zolgensma, and related news stories. We also look at some of our recent columns and forum posts, and share our thoughts. Listen here, and be sure to subscribe to the podcast. Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful information about gene therapy, including its history and how it is being investigated for the treatment of genetic diseases. Visit www.exploregenetherapy.com

Zolgensma drug by Avexis brought into AustraliaRoche - https://www.roche.com/research_and_development/what_we_are_working_on/neuroscience/approaching-sma.htmParliament date to be announced, looks like some time in August 2019 (Awareness Month)Gala in Melbourne and Brisbane. Purchase tickets here: https://smaaustralia.org.au/get-involved/upcoming-eventsPlease contact us if you would like to see a Sydney eventBe SMA Aware App currently in development. Please share what are things that you are struggling with and need support with.Currently working on a dedicated SMA Conference for May 2020 (potentially on the Gold Coast)Newborn Screening fact sheet:https://www.facebook.com/SpinalMuscularAtrophyAustralia/photos/a.370736359243/10157422001809244/?type=3&theater

When scientists are able to develop a new treatment for a rare disease that severely impacts the lives of very young children, it’s generally considered good news.And in the case of the newest announcement from Novartis, it is. But it’s not that simple.The drugmaker, who is parent to the company AveXis, recently revealed a new treatment for a debilitating genetic disease called spinal muscular atrophy. The condition affects up to 25,000 people and is currently the top genetic cause of death for infants, and a new gene therapy has recently gained FDA approval to treat it. But it seems there’s a catch: the one-time treatment, called Zolgensma, comes with the largest price tag for any drug treatment in the world: $2.1 million.So, even in a country known for its skyrocketing prescription costs, is this defensible? Novartis CEO Vas Narasimhan believes the price tag is fair when you consider the tradeoff, stating "Zolgensma could create a lifetime of possibilities for the children and families impacted by this devastating condition.”Initially, the company publicly discussed pricing possibilities that could reach up to $5 million, which is why Dr. Steve Pearson, the president of the Institute for Clinical and Economic Review, is declaring this actual price “a positive outcome.” But while many experts acknowledge that treatment for this condition is already quite expensive, neither of these defenses have been enough to insulate the drug maker – and industry at large – from backlash.David Mitchell, founder of the advocacy group Patients For Affordable Drugs, released a statement Friday calling the situation “emblematic of our broken system.” And while Novartis suggests that an initial balloon payment for “a lifetime of benefits” is an improvement on the pay-as-you-go model for chronic illnesses, Mitchell is critical, saying: "We didn't pay for the polio vaccine based on the future cost savings for kids who didn't need to live in iron lungs."

In this special edition of the SMA News today podcast, Forums Editor Michael Morale discusses AveXis' recent announcement that experimental SMA gene therapy AVXS-101 has been submitted to the US Food and Drug Administration (FDA) for approval.

AveXis submits AVXS-101 to FDA.

1. Featured Article: Nusinersen in spinal muscular atrophy type 1 patients older than 7 months: a cohort study2. What’s Trending: MRI-guided thrombolysis for stroke with unkown time of onset (WAKE-UP study)In the first segment, Dr. Ted Burns talks with Dr. Laurent Servais about his paper on safety and efficacy of nusinersen treatment in patients with SMA type 1 older than 7 months. In the second part of the podcast, we are featuring a discussion with Dr. Jim Siegler and Dr. Christian Gerloff on the results of the WAKE-UP stroke trial.DISCLOSURES: Dr. Servais has served as scientific consultant in the S.A.B of Roche, Biomarin, aTyr Pharma, Dynacure, Biogen, Bamboo, Sarepta, and Avexis; has received speaker honoraria gave lecture sponsored by Biomarin, Biogen, and Sarepta; holds patents as co-inventor of the Moviplate device, and of Actimyo; has consulted for Bamboo therapeutics, Pfizzer, Anagenesis, Avexis, and Dynacure; has received research support from NHS, Valerion, Dynacure, Avexis, Biogen, Roche, FP7 programs Skip and Scope, Université de Liège, and Association Française contre les Myopathies.

PharmaPills - Pillole dal farmaceutico: Novità, Curiosità e Lavoro dal mondo del farmaceutico. A cura di Stefano LagravineseIn questa puntata parliamo di:Aziende: Novo Nordisk, MolMed, Orchard Therapeutics, Gsk, Astrazaneca, Recordati, Mylan, Orphan Europe, Eli Lilly, Sigilon Therapeutics, Farmindustria, AveXis, Novartis, Leo Pharma Italia Persone: Lorenzo Wittum (Astrazaneca Italia), Eugenio Aringhieri (Farmindustria)Nuove terapie: Cisteamina bitartratoPatologie: Cistinosi nefropatica, diabete, atrofia muscolare spinale (SMA), psoriasi, tumori testa-collo.Lavoro: Clinical Trial Associate, Clinical Research Associate Ogni mercoledì alle h 12.00 su Spreaker.com e iTunes.Seguici su: www.telegram.me/pharmapillswww.facebook.com/pharmapills/Hai un dispositivo Apple? Seguici e abbonati al podcast tramite la app iPod http://nelfarmaceutico.link/pharma-apple

PharmaPills - Pillole dal farmaceutico: Novità, Curiosità e Lavoro dal mondo del farmaceutico. A cura di Stefano LagravineseIn questa puntata parliamo di:Aziende: Novo Nordisk, MolMed, Orchard Therapeutics, Gsk, Astrazaneca, Recordati, Mylan, Orphan Europe, Eli Lilly, Sigilon Therapeutics, Farmindustria, AveXis, Novartis, Leo Pharma Italia Persone: Lorenzo Wittum (Astrazaneca Italia), Eugenio Aringhieri (Farmindustria)Nuove terapie: Cisteamina bitartratoPatologie: Cistinosi nefropatica, diabete, atrofia muscolare spinale (SMA), psoriasi, tumori testa-collo.Lavoro: Clinical Trial Associate, Clinical Research Associate Ogni mercoledì alle h 12.00 su Spreaker.com e iTunes.Seguici su: www.telegram.me/pharmapillswww.facebook.com/pharmapills/Hai un dispositivo Apple? Seguici e abbonati al podcast tramite la app iPod http://nelfarmaceutico.link/pharma-apple

-Novartis buys gene therapy company, Avexis for $8.7 billion http://investors.avexis.com/phoenix.zhtml?c=254285&p=irol-newsArticle&ID=2341482-Spark is another gene therapy company I like, involved in hemophilia and eye-related blindness diseases http://sparktx.com/Follow me on twitter @matthewlepoire

Biogen misses out on a huge deal that Novartis completed with AveXis. Plus, Incyte's phase 3 implosion, and what it means for immuno-oncology more broadly.

Today in FirstWord:

Here’s your Headstart on the latest business headlines for Monday April 9th, 2018. Coming up:Novartis enters in an agreement to acquire AveXis for $8.7 BillionDeutsche Bank Appoints a New CEOPayPal Expands to KenyaAmerican Airlines Purchases 47 New 787 Boeing JetsCoinbase Looks to Potentially Become a Regulated ExchangeComcast Partners with the Chicago Cubs in a Multi-year DealAmazon Looks to Potentially Offer Person-to-Person PaymentsWalmart Rolls Out eCommerce Vending MachinesAlibaba Invests in SenseTime, China’s Leading Artificial Intelligence CompanyAdidas Plans to Shut Down the Majority of its Stores in an Effort to Focus on its Digital Business See acast.com/privacy for privacy and opt-out information.

Imagina você dormir ontem e acordar hoje com 78% de valorização sem precisar de fazer nada, sem nem entender suas ações que antes no inicio do ano eram cotadas a $93 hoje amanhecem a $211. Um ganho fenomenal. Parece algo impossível? Para os acionistas da Avexis não! Eles receberam uma proposta de compra da Novartis […] The post Papo de Dividendo #42: Novartis – PYPL – FB – BCS – MGM – WYNN appeared first on Viver de Dividendos.

AveXis to Begin Pivotal AVXS-101 Clinical Trial for SMA: https://smanewstoday.com/2017/10/03/fda-approves-pivotal-trial-of-avxs-101-for-sma-type-1/ A List of Great SMA Books: https://smanewstoday.com/2017/09/28/four-books-by-sma-authors/

Show description/summary:1) Practice guideline summary: Sudden unexpected death in epilepsy 2) What's Trending: 21st Century Cures ActThis podcast begins and closes with Dr. Robert Gross, Editor-in-Chief, briefly discussing highlighted articles from the April 25, 2017 issue of Neurology. In the first segment, Dr. Andrew Schomer interviews Dr. Cynthia Harden about the AAN practice summary guideline on sudden unexpected death in epilepsy (SUDEP). Then, for our “What's Trending” feature of the week, Dr. Jason Crowell talks with Dr. Nicholas Johnson about the 21st Century Cures Act. Disclosures can be found at Neurology.org.DISCLOSURES: Dr. Johnson serves as Associate Editor for Neurology: Genetics; receives research support from Ionis Pharmaceuticals, Biogen Idec, Balerion Therapeutics, Cytokinetics, Acceleron, NINDS (grant 1K23NS091511-01), Muscular Dystrophy Association, and Myotonic Dystrophy Foundation; and consults for AMO Pharma and AveXis. Dr. Schomer, Harden, and Crowell report no disclosures.