548 Epizód

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

    Közzétéve: 2023. 03. 03.

  2. A Next-Generation RNA Therapy Targets Telomere Disorders

    Közzétéve: 2023. 03. 03.

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

    Közzétéve: 2023. 02. 24.

  4. Translating Urgency into Science

    Közzétéve: 2023. 02. 16.

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

    Közzétéve: 2023. 02. 10.

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

    Közzétéve: 2023. 02. 02.

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

    Közzétéve: 2023. 01. 26.

  8. Powering Cells in People with Rare Mitochondrial Diseases

    Közzétéve: 2023. 01. 19.

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

    Közzétéve: 2023. 01. 12.

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

    Közzétéve: 2023. 01. 05.

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

    Közzétéve: 2022. 12. 29.

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

    Közzétéve: 2022. 12. 22.

  13. Treating Rare Endocrine Disorders with Therapeutic Peptides

    Közzétéve: 2022. 12. 15.

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

    Közzétéve: 2022. 12. 08.

  15. Addressing the Current Limitations of AAV Gene Therapies

    Közzétéve: 2022. 12. 01.

  16. Examining the Legislative Landscape for Rare Disease Drug Development

    Közzétéve: 2022. 11. 24.

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

    Közzétéve: 2022. 11. 18.

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

    Közzétéve: 2022. 11. 10.

  19. Addressing Racial Disparities in a Rare Blood Cancer

    Közzétéve: 2022. 11. 03.

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

    Közzétéve: 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.

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