Rare disease science with human stakes.
RARECast treats rare disease as a medical, scientific, and policy problem with human stakes. Its interviews move patiently from diagnosis and daily burden to clinical trials, data strategy, drug development, and regulatory decisions.
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RARECast is a long-running interview show focused on rare disease medicine, research, advocacy, and policy. It has a calm, explanatory style. Episodes do not chase novelty for its own sake; they tend to ask what a condition does, what current care misses, and what new science might realistically change. Recent conversations range from functional precision medicine in rare pediatric cancer to oral medicines for rare autoimmune diseases, parent-led gene therapy efforts, antisense programs for Schaff-Yang Syndrome, and the burden of polycythemia vera. The show also spends time on policy problems that shape care, including data collection, patient engagement, newborn screening, clinical trial endpoints, and regulatory approval paths. Its medical vocabulary is substantial. Genomics, AAV9 gene replacement, antisense oligonucleotides, lactic acidosis, MECP2 expression, and minimum data sets appear as working terms rather than decorative jargon. Still, the interviews usually connect those terms to concrete consequences. A therapy is discussed in terms of hand function, breathing, feeding, survival, blood counts, toxicities, or the ability of families to be seen by systems that often overlook them. The show’s emotional center is restrained. It can cover the death of a child, lifelong caregiving, experimental treatment, and parent-built foundations without turning those stories into spectacle. That restraint is part of its usefulness. Guests are given room to explain how research moves from cell and animal models toward first-in-human studies, how families fund science when no one else will, and why regulators or payers may not measure the outcomes patients value most. The result is a show that sits between medical journalism, patient advocacy, biotech briefing, and health policy seminar. It is not light background listening. It rewards attention, especially from people tracking rare disease treatments, advocacy organizations, clinical development, or the practical friction between promising science and actual care.
Made for: For rare disease families, advocates, clinicians, researchers, biotech watchers, and policy-minded listeners who want careful context rather than quick takes. It also suits people following gene therapy, precision medicine, patient data, and clinical trial design.
What sets it apart: RARECast stands out by linking medical mechanisms and treatment programs to family experience, regulatory pressure, and health-system decisions. It gives technical guests room to explain while keeping patient burden and caregiver knowledge central.
RARECast is a Global Genes podcast hosted by award-winning journalist Daniel Levine. It focuses on the intersection of rare disease with business, science, and policy.
Based on 6 episodes we listened to · September 2026
RARECast makes rare-disease science, treatment development, genetic counseling, and regulatory conflict legible without sanding away their complexity.
RARECast follows rare-disease care into the rooms where consequential decisions are made. Daniel Levine uses focused interviews to clarify how experimental therapies work, how clinical evidence is judged, and how policy shapes access.
A gene therapy gets one shot; Daniel Levine asks how it reaches the right cells.
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Short reviews from the PlayNext desk, based on the episodes we processed.
A serious case for matching cancer treatment to the child, not the average.
Jim Foote's story gives the conversation its emotional center, shaped by the loss of his son Trey to osteosarcoma and by the turn toward First Descent Biomedical's functional precision medicine work for rare and pediatric cancers.
A careful look at how trial results can miss what families live with every day.
Rarecast follows families of children with the ultra-rare mitochondrial disorder pyruvate dehydranase complex deficiency, or PDCD, as they push the FDA toward a path to approval for saltherapeutics experimental therapy without another trial.
A grounded RareCast episode on NGN-401, Rett syndrome, and the challenge of restoring MECP2 safely.
RareCast frames Rett syndrome through the daily realities it can reshape: purposeful hand use, communication, movement, and the long caregiving arc families carry.
A measured case for treating rare-disease data as health infrastructure.
Rare diseases are often discussed through cost and strain, but this Rarecast conversation keeps its attention on what better data might unlock.
A careful look at how genetic testing can turn one private diagnosis into a family question.
A single genetic diagnosis can quickly widen into a family matter. RareCast follows Daniel Levine's conversation with Rajani Atre, Senior Genetic Counselor at Michigan Medicine's Frankel Cardiovascular Center, on cascade genetic testing: targeted testing for biological relatives…
A focused RareCast discussion on FGFR3 selectivity and Tyra's experimental oral achondroplasia therapy.
Achondroplasia gets a tightly focused RareCast discussion through Todd Harris, CEO of Tyra Biosciences, who frames the condition around genetic diagnosis, bone growth, and drug development.
A measured episode on how BTK research connects rare-disease symptoms with mechanism-driven care.
Rare autoimmune diseases can seem separate at first glance, but Rarecast keeps its focus on the biology they may share. Daniel Levine talks with Pablo Sardi, head of rare disease research at Sanofi, about rilzabrutinib, an oral BTK inhibitor, and the push to match treatment with…
Measured MS science, translated for care.
Clinical research, translated for practice.
Serious conversations about better health care.
Daniel Levine interviews Pablo Sardi, head of rare disease research at Sanofi, about the company's oral BTK inhibitor rilzabrutinib.
This episode discusses Acadia Pharmaceuticals' approval of DEBU, the first treatment for Rett syndrome, and their ongoing pipeline development for additional therapies.
This episode discusses the FDA approval of papzimios, an immunotherapy for recurrent respiratory papillomatosis, featuring insights from a foundation president and a medical expert.
Katrina Rice discusses how her personal experience as a mother of a child with sickle cell disease informs her work in clinical trials to improve engagement with rare disease participants.
Daniel Levine interviews Todd Harris, CEO of Tyra Biosciences, about their experimental oral drug Tyra 300 for achondroplasia.
Monica DeBoe spent 20 months and 111 appointments with 26 specialists before a digital motion X-ray led to a craniocervical-instability diagnosis.
Jim Foote traces First Descent Biomedical back to the loss of his son Trey to osteosarcoma.
This episode explores Sanofi's research into using a single oral therapy to treat multiple rare autoimmune diseases by targeting root causes rather than just symptoms.
This episode features an interview with Shpila Morozovich, a researcher and mother who founded the CTNNB1 Foundation to develop a gene therapy for her son's ultra-rare neurodevelopmental disorder, discussing her journey from diagnosis to clinical trials.
This episode explores how the Foundation for Prader-Willi Research's Genesys Initiative is developing a drug program using antisense oligonucleotide technology to treat Schaff-Yang Syndrome, an ultra-rare neurodevelopmental disorder.
This episode explores polycythemia vera, a rare blood cancer, discussing its impact on patients' lives and promising new treatments in development.
Rarecast turns to the fight over pyruvate dehydranase complex deficiency, or PDCD.
Rett syndrome is the starting point.
Rarecast's interview with Will Green centers on the World Economic Forum paper "Making Rare Diseases Count" and its argument that better rare disease data can unlock a multi-trillion dollar opportunity.
Rarecast's Daniel Levine speaks with Rajani Atre, Senior Genetic Counselor at Michigan Medicine's Frankel Cardiovascular Center, about cascade genetic testing after one family member gets a disease-causing variant.
This episode features an interview with Todd Harris, CEO of Tyra Biosciences, discussing the company's experimental once-daily oral medicine for achondroplasia, its competitive advantages over existing therapies, and insights from studies conducted to date.
This episode discusses the FDA approval of Presagen's papzimios immunotherapy for treating recurrent respiratory papillomatosis, a rare respiratory disease caused by HPV.