548 Episodo

  1. Overcoming a Delivery Challenge of Retinal Gene Therapies

    Publicado: 19/6/2025

  2. A Gene Editing First Augurs an Era of Bespoke Therapies

    Publicado: 12/6/2025

  3. How One Patient Organization Drives Drug Development

    Publicado: 5/6/2025

  4. Using CRISPR to Modulate Gene Expression

    Publicado: 29/5/2025

  5. Satisfying the Hunger for a Prader-Willi Therapy

    Publicado: 22/5/2025

  6. How an Academic Medical Center Helped Change the Landscape for a Rare Disease

    Publicado: 15/5/2025

  7. Improving Outcomes for People with a Set of Rare Cancers

    Publicado: 8/5/2025

  8. An Effort to Build a Better DMD Gene Therapy

    Publicado: 2/5/2025

  9. A Once-Failed Pain Therapy Shows Potential in a Neurodevelopmental Disorder

    Publicado: 24/4/2025

  10. Advancing a Cutting-Edge Therapy for a Rare, Childhood Cancer

    Publicado: 17/4/2025

  11. Learning to Take His Vitamins

    Publicado: 10/4/2025

  12. The First Treatment for a Rare Neurodegenerative Condition Awaits FDA Approval

    Publicado: 3/4/2025

  13. Engineering Skin Bacteria to Be Live Biotherapeutics

    Publicado: 27/3/2025

  14. Seeing the Gene and Cell Therapy Translational Divide as an Opportunity

    Publicado: 20/3/2025

  15. My Mother, Myself, and ALS

    Publicado: 13/3/2025

  16. From Immovable Object to Advocacy Force

    Publicado: 6/3/2025

  17. A Rapid and Scalable Approach for Screening Personalized ASOs

    Publicado: 27/2/2025

  18. Addressing the Disease Mechanism of a Rare Kidney Disease

    Publicado: 20/2/2025

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

    Publicado: 13/2/2025

  20. A First for Rett Syndrome with More in the Pipeline

    Publicado: 6/2/2025

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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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