RARECast

Mar 06 2025 533 ep. 25 mins 181

RARECast Podcast artwork

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.

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From Immovable Object to Advocacy Force

Mar 06 2025 24 mins

Russ Walter, liaison and board member of the GBS-CIDP Foundation International, discusses his recovery from Guillian-Barre syndrome, the changing medical landscape for the condition, and why his experience led him to become a patient advocate.

A Rapid and Scalable Approach for Screening Personalized ASOs

Feb 27 2025 23 mins

Scott Younger, director of disease gene engineering at Children Mercy Kansas City’s Genomic Medicine Center, discusses a process he developed to rapidly and inexpensively create organoids from a person’s induced pluripotent stem cells, how the process was used to match three children with Duchenne muscular dystrophy to an antisense oligonucleotide therapy, and the potential imp

Addressing the Disease Mechanism of a Rare Kidney Disease

Feb 20 2025 25 mins

Marshall Fordyce, founder and CEO of Vera Therapeutics, discusses IgA nephropathy, the company’s experimental therapy atacicept, and its potential in IgAN other B cell-mediated diseases.

The Mother of a Son with SCD, Applies Direct Experience to Her Clinical Trials Work

Feb 13 2025 38 mins

Katrina Rice, chief delivery officer of biometrics services at the clinical trial data management and analytics company eClinical Solutions, discusses her experience as a mother of a child with a rare disease has informed her professional life, and how she has used that to educate her colleagues and clinical trial sponsors about how to better engage with rare disease clinical trial

A First for Rett Syndrome with More in the Pipeline

Feb 06 2025 23 mins

Elizabeth Thompson, executive vice president and head of research and development for Acadia, discusses Rett syndrome, how Daybue has changed the treatment landscape for people with the condition, and the company’s advancing pipeline to treat it.

A Heart Attack that Led to a Rare Disease Diagnosis

Jan 30 2025 30 mins

Patty Keating, executive director of the HypoPARAthyroidism Association, discusses her experience with hypoparathyroidism, why it can be challenging to diagnose, and the changing treatment landscape.

Targeting Rare Liver Diseases with Gene Editing Therapies

Jan 23 2025 23 mins

Joe Truitt, CEO of iECURE, discusses the company’s experimental in vivo gene-editing therapy for OTC, how it works, and its the complete response seen in the first infant treated with the medicine.

Using AI to De-Risk Rare Disease Drug Development

Jan 16 2025 43 mins

Tim Guilliams, co-founder and CEO of Healx, discusses the origin of the company, its lead asset in development for neurofibromatosis type 1, and how he views the company’s progress to date.

Addressing a Growing Demand for Plasma-Derived Therapies

Jan 09 2025 30 mins

Joerg Schuettrumpf, chief scientific innovation officer at Grifols, discusses the changing landscape for primary immunodeficiency disorders, the challenges of ensuring a reliable supply of plasma-derived therapies, and how treatment options may evolve.

Empowering Patients with Data to Drive Drug Development

Jan 02 2025 43 mins

Citizen Health Co-Founders Farid Vij and Nasha Fitter discuss the evolution of the rare patient data platform Citizen Health, what it will enable, and how it is helping shift power towards patients and their caregivers in the pursuit of new therapies.