# rare disease (audio feeds) — RSS Amplifier Recent posts from the 4 feeds in the RSS Amplifier directory that cover rare disease. Page: Feed: --- ## Curing Sickle Cell Before Life Begins _2026-08-13 · RARECast_ Sickle cell disease is caused by a single mutation in the beta-globin gene that leads to painful crises, anemia, and organ damage. Despite advances in treatment, it remains a devastating and often overlooked global health challenge, particularly in low-resource settings where children frequently go undiagnosed until life-threatening complications arise. The PERICLES project is an ambitious… [Listen](https://rss.art19.com/episodes/8ed1a96a-f60e-4ce9-b441-8ed3c53c8065.mp3?rss_browser=BAhJIhRSU1NBbXBsaWZpZXJCb3QGOgZFVA%3D%3D--6345a2e4a0991d4cbe92da92e0fa95ff9d12de2e) ## Spotting Neuromuscular Red Flags _2026-08-06 · RARECast_ Many older adults may dismiss dropping objects, struggling with stairs, or tiring on short walks as a matter of just getting older, but it can be an early sign of a serious group of disorders known as late-onset neuromuscular diseases (LONDs). The American Neuromuscular Foundation’s Why Behind Your Weakness campaign seeks to raise awareness of LONDs and the shared symptom patterns that too often… [Listen](https://rss.art19.com/episodes/e4ca370d-4c65-4723-a10a-8974e4e15747.mp3?rss_browser=BAhJIhRSU1NBbXBsaWZpZXJCb3QGOgZFVA%3D%3D--6345a2e4a0991d4cbe92da92e0fa95ff9d12de2e) ## Expanding into a Global Rare Disease Player through Deal-Driven Innovation _2026-07-30 · RARECast_ Chiesi Global Rare Diseases has rapidly evolved from a small, regional rare‑disease business into a global organization, leveraging strategic deals and development bets to reshape standards of care for patients worldwide. Giacomo Chiesi, head of the unit, discusses how the business has grown through acquisitions, its move into CRISPR gene editing and blood–brain barrier‑crossing enzyme platforms,… [Listen](https://rss.art19.com/episodes/e4ec8a3c-c76e-41e4-9d3e-e0261673f219.mp3?rss_browser=BAhJIhRSU1NBbXBsaWZpZXJCb3QGOgZFVA%3D%3D--6345a2e4a0991d4cbe92da92e0fa95ff9d12de2e) ## When Geography, Cost, and Policy Become as Life-Limiting as a Disease _2026-07-23 · RARECast_ When Tom Sayiner was diagnosed with the fatal neurodegenerative disease ALS, he and his wife, Tamara, learned that tofersen had been approved in Europe as a therapy that could slow the progression of his genetic form of the disease. But the Sayiners, who live in Sweden, soon discovered they could not access the drug because a Swedish health technology assessment council determined there was… [Listen](https://rss.art19.com/episodes/ba4cd802-900a-4076-9c2b-370821734baa.mp3?rss_browser=BAhJIhRSU1NBbXBsaWZpZXJCb3QGOgZFVA%3D%3D--6345a2e4a0991d4cbe92da92e0fa95ff9d12de2e) ## A One-Time Cell Therapy to Reset the Immune System in Autoimmune Diseases _2026-07-16 · RARECast_ People with rare, severe autoimmune diseases often live for years with progressive, disabling conditions managed by chronic immunosuppression that rarely addresses the underlying cause. Kyverna is developing an autologous CAR T-cell therapy designed to deliver a deep immune reset by broadly depleting pathogenic B cells, followed by repopulation with naïve, non-pathogenic B cells and normalization… [Listen](https://rss.art19.com/episodes/315ac594-9982-4a63-b6e1-1f38273d97f3.mp3?rss_browser=BAhJIhRSU1NBbXBsaWZpZXJCb3QGOgZFVA%3D%3D--6345a2e4a0991d4cbe92da92e0fa95ff9d12de2e) ## Building the Infrastructure for Made‑to‑Order Gene Therapies _2026-07-09 · RARECast_ The case of baby KJ Muldoon, an infant born with a lethal genetic metabolic disorder, demonstrates the potential to compress years of therapeutic development into months using an in vivo base editing approach. Jeff Coller, director of the Johns Hopkins RNA Innovation Center, wrote about the case in a New York Times op-ed, arguing that CRISPR-based base editors—delivered via lipid nanoparticles as… [Listen](https://rss.art19.com/episodes/c04217ff-31b9-49bd-a9db-6029e3be8d3b.mp3?rss_browser=BAhJIhRSU1NBbXBsaWZpZXJCb3QGOgZFVA%3D%3D--6345a2e4a0991d4cbe92da92e0fa95ff9d12de2e) ## Rewiring the Rare Disease Diagnostic Odyssey _2026-07-02 · RARECast_ Families seeking a diagnosis for a rare disease often face a protracted diagnostic odyssey that can include ER visits, specialist referrals, and dead ends, even at world-class medical centers. Parents bounce from doctor to doctor while payers absorb mounting costs, and the pivotal moment of putting a name to a disease—which can reduce unnecessary care and emotional distress—arrives late, if at… [Listen](https://rss.art19.com/episodes/59915023-9190-40c1-b6e1-3fca2bdc2f36.mp3?rss_browser=BAhJIhRSU1NBbXBsaWZpZXJCb3QGOgZFVA%3D%3D--6345a2e4a0991d4cbe92da92e0fa95ff9d12de2e) ## Emerging from a Life in the Shadows _2026-06-25 · RARECast_ Erythropoietic protoporphyria (EPP) is a rare, inherited metabolic disorder that triggers a toxic photochemical reaction in skin when exposed to light. A short time in the sun for someone with the condition can result in excruciating, second-degree–like burns and leaves patients sidelined from normal outdoor activity. Craig Leppert, who has EPP, saw his childhood shaped by constant vigilance… [Listen](https://rss.art19.com/episodes/6553f69c-d236-49a9-bf5d-c20f93c38c06.mp3?rss_browser=BAhJIhRSU1NBbXBsaWZpZXJCb3QGOgZFVA%3D%3D--6345a2e4a0991d4cbe92da92e0fa95ff9d12de2e) ## Fitting Big Genes into Small Vectors _2026-06-18 · RARECast_ Many disease‑causing genes are too large to be packaged into standard AAV gene therapy vectors, leaving a long list of otherwise gene-therapy-ready conditions without viable treatments. SpliceBio is leveraging a protein splicing platform based on engineered split inteins to overcome the cargo limitations of AAV gene therapy vectors, enabling delivery of large genes to potentially treat a broad… [Listen](https://rss.art19.com/episodes/a15f067f-1fb9-44f0-b55a-7caf1c75ccce.mp3?rss_browser=BAhJIhRSU1NBbXBsaWZpZXJCb3QGOgZFVA%3D%3D--6345a2e4a0991d4cbe92da92e0fa95ff9d12de2e) ## Targeting Iron Dysregulation in the Neurodegenerative Condition MSA _2026-06-11 · RARECast_ Multiple system atrophy is a rapidly progressive neurodegenerative condition that is often misdiagnosed as Parkinson’s disease but carries a far grimmer prognosis. MSA has a median survival of just seven to eight years after symptom onset. Toxic aggregates of alpha‑synuclein and excess brain iron create a vicious cycle of neuronal damage that drives the multisystem motor and autonomic decline… [Listen](https://rss.art19.com/episodes/a6c068d5-76bc-4ae4-937e-5fd3cf614ae7.mp3?rss_browser=BAhJIhRSU1NBbXBsaWZpZXJCb3QGOgZFVA%3D%3D--6345a2e4a0991d4cbe92da92e0fa95ff9d12de2e) ## [8.1% APY Plus Free Stock (Sponsored)](https://crawlproof.com/a/7Dbgvbr0iwoV) _2026-06-11 · **Sponsored**_ Claim 8.1% APY and up to $1,030 in SK hynix and Nvidia stock. ## [300 - Season Finale: 300 Episodes Rooted in Connection](https://twodisableddudes.com/300-season-finale-300-episodes-rooted-in-connection/) _2026-05-18 · Two Disabled Dudes_ In this special 300th episode of the Two Disabled Dudes Podcast, Sean and Kyle reflect on nearly 10 years of podcasting, the challenges of creating 300 episodes, and the incredible community that has formed along the way. From forgotten recordings and technical disasters to meaningful live events and lifelong friendships, they share stories from behind the scenes and revisit some of their favorite… [Listen](https://episodes.captivate.fm/episode/96c5a0da-95b2-46af-85a7-525a93b18e94.mp3) ## [299 - Building Hope From Broken Systems](https://twodisableddudes.com/299-building-hope-from-broken-systems/) _2026-05-11 · Two Disabled Dudes_ Sean and Kyle kick off this episode with a conversation about smart technology, AI, and the growing tension between convenience and commercialization in everyday life. That discussion sets the stage for a powerful interview with Joshua Resnikoff , founder and CEO of Sunstone Health , whose family’s rare disease journey inspired him to tackle one of healthcare’s biggest challenges: the long and… [Listen](https://episodes.captivate.fm/episode/26bbe4dd-a797-48a4-8224-157fc8d7e08f.mp3) ## [298 - Invisible Illness, Unbreakable Bond: Part 2](https://twodisableddudes.com/298-invisible-illness-unbreakable-bond-part-2/) _2026-05-04 · Two Disabled Dudes_ Part 2 continues Debbie Drell’s story, focusing on the real-life impact of caregiving, invisible illness, and the need for empathy. Debbie highlights how much people take for granted—walking short distances, being in crowds, traveling, or getting through a full day without rest. For her sister Alex, who lives with multiple rare conditions including pulmonary hypertension, these everyday activities… [Listen](https://episodes.captivate.fm/episode/24561a74-bc8e-4658-9d0c-aadbdb40d86e.mp3) ## [Solving Patient Matching in Rare Disease Trials](https://podcasters.spotify.com/pod/show/rare-diseases-roundup/episodes/Solving-Patient-Matching-in-Rare-Disease-Trials-e3ikuml) _2026-04-29 · Partners4Access · P4A Let's Talk Rare: The Life Science Podcast_ One of the most persistent barriers in rare disease drug development isn’t the science — it’s identifying and reaching the small number of patients who could benefit from emerging therapies. Without solving this, even the most promising innovations risk never reaching the people who need them most. In our latest podcast episode, we explore how the industry can better connect patients to clinical… [Listen](https://anchor.fm/s/47fe5f0/podcast/play/119224469/https%3A%2F%2Fd3ctxlq1ktw2nl.cloudfront.net%2Fstaging%2F2026-3-29%2F4adba2de-76d1-9e64-1bb0-b05bd5959d49.mp3) ## [297 - Invisible Illness, Unbreakable Bond: Part 1](http://twodisableddudes.com) _2026-04-27 · Two Disabled Dudes_ In this first part of the conversation, Sean and Kyle sit down with longtime friend and rare disease advocate Debbie Drell, whose connection to the space runs deep—both professionally and personally. Debbie shares the story of her sister Alex, who was diagnosed with pulmonary hypertension decades ago and given just two years to live—yet is still fighting today. As both a caregiver and advocate,… [Listen](https://episodes.captivate.fm/episode/212633b7-3e3c-4366-824b-1ed07f64544e.mp3) ## [296 - Support That Meets You Where You Are](https://twodisableddudes.com/296-support-that-meets-you-where-you-are/) _2026-04-20 · Two Disabled Dudes_ Episode 296 explores connection, resilience, and the power of meeting people where they are—starting with a story about a young man with Friedreich’s ataxia (FA) who courageously continues cycling even after a crash. The episode’s main conversation features Dr. Eric Mitchell and his son Dylan, who share their work with Neurodiversity Consultants and their strengths-based, relationship-driven… [Listen](https://episodes.captivate.fm/episode/b86e6c37-ed26-4253-aed7-2708a7242eba.mp3) ## [295 - Pushing Harder Isn’t Always the Answer](http://twodisableddudes.com) _2026-04-13 · Two Disabled Dudes_ Another frustrating hotel experience opens the episode, but the real conversation centers on what it actually means to “never give up.” After dealing with a series of accessibility failures—miscommunication, poor accommodations, and a lack of understanding—Kyle and Sean shift into a bigger idea: persistence isn’t just about pushing harder. Using the story of a breakthrough Friedreich’s ataxia (FA)… [Listen](https://episodes.captivate.fm/episode/40529270-7752-47da-98ef-e071eb5d328e.mp3) ## [294 - Finding Your People Without Losing Yourself](http://twodisableddudes.com) _2026-03-30 · Two Disabled Dudes_ This episode starts with a classic 2DD moment—a wild but relatable story about Kyle discovering he had a piece of road stuck in his knee for a year and a half. It’s funny, a little absurd, and quickly turns into something more meaningful: how often we ignore small issues—physical or otherwise—and just learn to live with them instead of addressing them. From there, the conversation shifts into a… [Listen](https://episodes.captivate.fm/episode/899972a6-b861-4abd-91c1-2f0e9a85e33a.mp3) ## [293 - Rare Disease Day: This is What Advocacy Looks Like](http://twodisableddudes.com) _2026-03-23 · Two Disabled Dudes_ In this special Rare Disease Day conversation, The Dudes partner with Jett Foundation to moderate a thoughtful panel on advocating for care needs, independence, and life with disability. Joined by Jake, Ashley, Charlie, and Xavier, the discussion highlights the many forms advocacy can take—from managing care and navigating insurance to simply showing up in the world, pursuing work, and building a… [Listen](https://episodes.captivate.fm/episode/cd350609-1aad-4117-b744-1b33a764dc8f.mp3) ## [292 - The Hidden System Behind Rare Disease Treatments](http://twodisableddudes.com) _2026-03-16 · Two Disabled Dudes_ What starts with airport chaos and accessibility frustrations turns into a meaningful conversation about what it really takes to move rare disease research forward. Sean and Kyle kick things off with travel stories from their trip to Rare At Sea , including misplaced mobility equipment, inaccessible hotel setups, and the all-too-common surprises that come with traveling disabled. Then they’re… [Listen](https://episodes.captivate.fm/episode/4f1214d9-3e05-497c-a4b3-04d4e3d8647f.mp3) ## [One platform, from silicon to agent (Sponsored)](https://crawlproof.com/a/NCZZqUDBu27t) _2026-03-16 · **Sponsored**_ Serverless inference plus Inference Router to optimize cost and latency at scale. ## [291 - Taking on the World’s Longest Stairway](http://twodisableddudes.com) _2026-03-09 · Two Disabled Dudes_ In this episode, Sean shares the bold details behind his upcoming climb of the Niesen Stairway in Switzerland—the world’s longest staircase with 11,674 steps, the equivalent of climbing the steps of the world’s tallest building nearly four times. Because the stairs are a private emergency access route alongside a mountain tram, Sean and his team will attempt the ascent overnight in the dark,… [Listen](https://episodes.captivate.fm/episode/a234c566-504c-4670-9c3d-9bb2470aa8c6.mp3) ## [Rare Disease Day Special: The stats and stories you need to hear.](https://podcasters.spotify.com/pod/show/rare-diseases-roundup/episodes/Rare-Disease-Day-Special-The-stats-and-stories-you-need-to-hear-e3fmdtf) _2026-02-27 · Partners4Access · P4A Let's Talk Rare: The Life Science Podcast_ Rare Disease Day Special | Let’s Talk Rare This Rare Disease Day, Let’s Talk Rare releases a special episode focused on the data and real-world context behind rare diseases. The episode features Iro Malekous , consultant at Partners4Access, who discusses: Key statistics shaping the rare disease landscape What those numbers mean for patients and families Gaps in diagnosis, access, and treatment Why… [Listen](https://anchor.fm/s/47fe5f0/podcast/play/116127087/https%3A%2F%2Fd3ctxlq1ktw2nl.cloudfront.net%2Fstaging%2F2026-1-27%2F29d3b49f-a6a4-4001-acd3-8adcb90596c3.mp3) ## [2026 Trends: A Year Of Change & Opportunity](https://podcasters.spotify.com/pod/show/rare-diseases-roundup/episodes/2026-Trends-A-Year-Of-Change--Opportunity-e3ed70j) _2026-01-30 · Partners4Access · P4A Let's Talk Rare: The Life Science Podcast_ 26 isn’t just another year in market access — it’s a turning point. In this episode of Let’s Talk Rare , P4A dives into the trends that are about to reshape how rare disease therapies reach patients across Europe — and trust us, the ground is moving. Policy is back in the spotlight. With the EU Pharmaceutical Strategy resurfacing, the big question is: How do we reward innovation and keep medicines… [Listen](https://anchor.fm/s/47fe5f0/podcast/play/114776531/https%3A%2F%2Fd3ctxlq1ktw2nl.cloudfront.net%2Fstaging%2F2026-0-30%2F9da5038d-a70e-e543-7377-e583bc5e9610.mp3) ## [Episode 52: Prolonged Seizures (Sponsored by UCB)](https://tsc-now.blubrry.net/2026/01/02/episode-52-prolonged-seizures-sponsored-by-ucb/) _2026-01-02 · TSC Now_ In a special episode of TSC Now, Dan interviews Bridgett Langstaff, mom to Jude, a 17-year-old living with tuberous sclerosis complex (TSC) and epilepsy. Bridgett shares Jude’s diagnosis story and how they ultimately made their way to Mass General Hospital and Dr. Thiele. She also talks about Jude’s ongoing struggle with seizures, from infantile spasms as a baby, to partial seizures, to tonic… [Listen](https://media.blubrry.com/tsc_now/media.blubrry.com/tsc_now/content.blubrry.com/tsc_now/UCB_Podcast_mixdown.mp3) ## [Going To Glasgow: ISPOR 2025](https://podcasters.spotify.com/pod/show/rare-diseases-roundup/episodes/Going-To-Glasgow-ISPOR-2025-e3boohr) _2025-12-02 · Partners4Access · P4A Let's Talk Rare: The Life Science Podcast_ In this episode of Let’s Talk Rare , we looked back at P4A’s recent trip to Glasgow, where our colleagues, Sam Morrison & Iro Malekou were on the ground for ISPOR activities leading up to ISPOR 2025 . They weren’t just observing—they were actively contributing , presenting a poster abstract , connecting with experts, and taking the pulse of what’s shaping the HEOR and rare disease landscape. We… [Listen](https://anchor.fm/s/47fe5f0/podcast/play/112009211/https%3A%2F%2Fd3ctxlq1ktw2nl.cloudfront.net%2Fstaging%2F2025-11-2%2F879a4337-6c46-3a09-b63b-c8561cb07d5c.mp3) ## [Episode 51: Drug-Resistant Epilepsy (Sponsored by LivaNova)](https://tsc-now.blubrry.net/2025/11/30/episode-51-drug-resistant-epilepsy-sponsored-by-livanova/) _2025-11-30 · TSC Now_ November is Epilepsy Awareness Month, and to help raise awareness, TSC Now host Dan Klein talks to Starr Phipps, an adult living with drug-resistant epilepsy. She shares her story, including how she faced stigma as a child, how her lack of control over her seizures impacted her emotional and physical well-being, and how continuing to push for treatments changed her life and inspired her to help… [Listen](https://media.blubrry.com/tsc_now/media.blubrry.com/tsc_now/content.blubrry.com/tsc_now/LivaNova_2025_Podcast_mixdown.mp3) ## [Conversations Beyond the Clinic](https://podcasters.spotify.com/pod/show/rare-diseases-roundup/episodes/Conversations-Beyond-the-Clinic-e3ae3ms) _2025-11-03 · Partners4Access · P4A Let's Talk Rare: The Life Science Podcast_ Recorded live from ESMO 2025, Conversations Beyond the Clinic explores the different perspectives of the attendees and stakeholders at the ESMO conference in Berlin. We asked a variety of medical stakeholders including A.I experts, nurses, researchers, patients and more to share their experiences of the conference and the work that brought them to ESMO. Through candid conversations we uncovers how… [Listen](https://anchor.fm/s/47fe5f0/podcast/play/110611612/https%3A%2F%2Fd3ctxlq1ktw2nl.cloudfront.net%2Fstaging%2F2025-10-3%2F9ed8cb17-3893-d4c9-341b-1d582c928bcf.mp3) ## [Voices Uncovered: Understanding Mental Health in Rare Diseases using Social Listening](https://podcasters.spotify.com/pod/show/rare-diseases-roundup/episodes/Voices-Uncovered-Understanding-Mental-Health-in-Rare-Diseases-using-Social-Listening-e38reqd) _2025-09-29 · Partners4Access · P4A Let's Talk Rare: The Life Science Podcast_ In this episode, we tune into the unfiltered voices of patients and caregivers navigating life with rare conditions. Through the power of social listening, we uncover the hidden struggles around mental health—stories often left unsaid in clinics but shared openly online. Joined by Lauren Roberts from Rareminds, we explore what these conversations reveal, why they matter, and how they can reshape… [Listen](https://anchor.fm/s/47fe5f0/podcast/play/108951821/https%3A%2F%2Fd3ctxlq1ktw2nl.cloudfront.net%2Fstaging%2F2025-8-29%2F19d6b6a9-7c25-d370-784a-9e47cfa7449a.mp3) ## [Stronger Together: Collaborating for Better Patient Information with Catherine Richards Golini](https://podcasters.spotify.com/pod/show/rare-diseases-roundup/episodes/Stronger-Together-Collaborating-for-Better-Patient-Information-with-Catherine-Richards-Golini-e37bi61) _2025-08-26 · Partners4Access · P4A Let's Talk Rare: The Life Science Podcast_ How do we make sure patients receive health information that is clear, trustworthy, and truly helpful? We sit down with Catherine Richards Golini from Karger Publishers to explore the power of collaboration in creating impactful patient resources. From working with healthcare professionals and patient advocates to partnering with researchers and publishers, Catherine shares how involving multiple… [Listen](https://anchor.fm/s/47fe5f0/podcast/play/107382401/https%3A%2F%2Fd3ctxlq1ktw2nl.cloudfront.net%2Fstaging%2F2025-7-26%2Fb2172e60-90b0-1fa2-f70e-7c62bf30e0df.mp3) ## [OST-HER2 and the Fight Against Osteosarcoma: A New Hope After 40 Years](https://podcasters.spotify.com/pod/show/rare-diseases-roundup/episodes/OST-HER2-and-the-Fight-Against-Osteosarcoma-A-New-Hope-After-40-Years-e34us43) _2025-07-01 · Partners4Access · P4A Let's Talk Rare: The Life Science Podcast_ Join us as we sit down with Paul Romness of OS Therapies to explore an exciting breakthrough in OST-HER2 targeted therapies for osteosarcoma —a rare and aggressive bone cancer. Learn how this innovative approach is offering new hope to patients, advancing precision medicine, and pushing the boundaries of rare disease treatment, not just for humans but for dogs too. Joining the discussion as always… [Listen](https://anchor.fm/s/47fe5f0/podcast/play/104869443/https%3A%2F%2Fd3ctxlq1ktw2nl.cloudfront.net%2Fstaging%2F2025-6-1%2F20a96112-a335-f33a-d712-de012c6f1d96.mp3) ## [Follow Releases, Get Reminders (Sponsored)](https://crawlproof.com/a/aPFBKenSZuZm) _2025-07-01 · **Sponsored**_ Follow a genre or a name and receive reminders on release day — free. ## [AI for Rare Disease: From Discovery to Trials, Smarter and Faster](https://podcasters.spotify.com/pod/show/rare-diseases-roundup/episodes/AI-for-Rare-Disease-From-Discovery-to-Trials--Smarter-and-Faster-e33h87f) _2025-05-29 · Partners4Access · P4A Let's Talk Rare: The Life Science Podcast_ Discover how artificial intelligence is transforming the future of rare disease research in the latest episode of Let’s Talk Rare , the official podcast from Partners4Access (P4A). Join us as we sit down with Ilya Burkov, AI expert at Nebius to explore how cutting-edge machine learning tools are accelerating the discovery, diagnosis, and development of treatments for rare conditions. With over 300… [Listen](https://anchor.fm/s/47fe5f0/podcast/play/103374511/https%3A%2F%2Fd3ctxlq1ktw2nl.cloudfront.net%2Fstaging%2F2025-4-29%2F4dd73bf9-45a1-743a-6de0-b47dc82f116d.mp3) ## [Improving Alignment In Drug Development](https://podcasters.spotify.com/pod/show/rare-diseases-roundup/episodes/Improving-Alignment-In-Drug-Development-e323l4o) _2025-04-28 · Partners4Access · P4A Let's Talk Rare: The Life Science Podcast_ In the latest episode of Let's Talk Rare, Owen Bryant & Georgie Rack delve into the world of drug development in rare diseases. They are joined by the brilliant Rob Freishtat, president of Uncommon Cures. Rob brings unique insights on who the key stakeholder are in developing drugs and the many ways that we can bring them together in order to improve and enhance the delivery of much-needed… [Listen](https://anchor.fm/s/47fe5f0/podcast/play/101880408/https%3A%2F%2Fd3ctxlq1ktw2nl.cloudfront.net%2Fstaging%2F2025-3-28%2Fd8fb1005-13d7-ac88-3ad4-2552aa0bb179.mp3) ## [Episode 50: What happens when a clinical trial fails?](https://tsc-now.blubrry.net/2025/04/11/episode-50-what-happens-when-a-clinical-trial-fails/) _2025-04-11 · TSC Now_ In the first episode of TSC Now in 2025, host Dan Klein tackles the challenging topic of what happens when a clinical trial fails and how failed clinical trials inform future research in tuberous sclerosis complex (TSC). Dan interviews Mary Kay Koenig, MD, Professor of Child Neurology and Associate Vice Chair for Clinical Research at University of Texas McGovern Medical School in Houston and… [Listen](https://media.blubrry.com/tsc_now/content.blubrry.com/tsc_now/Mary_Kay_Koenig_episode.mp3) ## [Navigating Housing Options for Dependent Adults with TSC (sponsored by Nobelpharma America)](https://tsc-now.blubrry.net/2024/12/24/navigating-housing-options-for-dependent-adults-with-tsc-sponsored-by-nobelpharma-america/) _2024-12-24 · TSC Now_ In this episode of TSC Now host Dan Klein is joined by Bruce Hainan and Jim Kotsailidis from Nobelpharma America, LLC and Shelly Meitzler, TSC Alliance Director of Outreach and Support, and mom to Ashlin and Mason with TSC. Shelly shares her experience looking for housing for her eldest daughter Ashlin, a dependent adult with tuberous sclerosis complex (TSC). She then shares the questions parents… [Listen](https://media.blubrry.com/tsc_now/content.blubrry.com/tsc_now/nobelpharma_pod_mixdown.mp3) ## [Drug-Resistant and Undertreated Epilepsy (sponsored by LivaNova)](https://tsc-now.blubrry.net/2024/11/27/drug-resistant-and-undertreated-epilepsy-sponsored-by-livanova/) _2024-11-27 · TSC Now_ In honor of National Epilepsy Awareness Month, TSC Now host Dan Klein is joined by Steven Wolf, MD, Director at the TSC Clinic Without Walls serving NYC, Westchester, Hudson Valley & Connecticut. Dr. Wolf also serves as the Director of Pediatric Epilepsy at Boston Children’s Health Physicians of New York and Connecticut. Dr. Wolf specializes in child neurology with a special emphasis on epilepsy.… [Listen](https://media.blubrry.com/tsc_now/content.blubrry.com/tsc_now/LivaNova_podcast_mixdown_FINAL.mp3)