548 Avsnitt

  1. How One Drugmaker Reaches out to Communities at Greater Risk for Rare Heart Condition

    Publicerades: 2023-03-03

  2. A Next-Generation RNA Therapy Targets Telomere Disorders

    Publicerades: 2023-03-03

  3. BridgeBio Advances Therapy for Limb-Girdle Muscular Dystrophy that Started with Two Patient Families

    Publicerades: 2023-02-24

  4. Translating Urgency into Science

    Publicerades: 2023-02-16

  5. Empowering Ultra-Rare Disease Patients to Pursue the Discovery of Treatments

    Publicerades: 2023-02-10

  6. Why a Topical Cannabidiol Gel May Help Treat the Behavioral Symptoms of Fragile X

    Publicerades: 2023-02-02

  7. An Ultra-Rare Disease Drug Developer Tries to Navigate Regulatory Uncertainty

    Publicerades: 2023-01-26

  8. Powering Cells in People with Rare Mitochondrial Diseases

    Publicerades: 2023-01-19

  9. Powering Weakened and Stressed Cells in ALS to Function Better with Nanocrystal Therapy

    Publicerades: 2023-01-12

  10. Developing a New Class of Therapies Based on a Natural Cargo Carrier

    Publicerades: 2023-01-05

  11. Reaching Beyond the Limits of Enzyme Replacement Therapies with Gene Therapies

    Publicerades: 2022-12-29

  12. One Woman’s Journey as a Caregiver to a Husband with Frontotemporal Dementia

    Publicerades: 2022-12-22

  13. Treating Rare Endocrine Disorders with Therapeutic Peptides

    Publicerades: 2022-12-15

  14. Tackling the Pricing Challenges for Advanced Therapies for Rare Diseases

    Publicerades: 2022-12-08

  15. Addressing the Current Limitations of AAV Gene Therapies

    Publicerades: 2022-12-01

  16. Examining the Legislative Landscape for Rare Disease Drug Development

    Publicerades: 2022-11-24

  17. How a Drug Setback Became a Patient Community’s Gain

    Publicerades: 2022-11-18

  18. Helping Regulators and Drug Developers Understand the Challenges of Living with Fabry Disease

    Publicerades: 2022-11-10

  19. Addressing Racial Disparities in a Rare Blood Cancer

    Publicerades: 2022-11-03

  20. How New Sequencing Technology Promises to Alter the Diagnostic Odyssey

    Publicerades: 2022-10-27

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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.

Visit the podcast's native language site