548 Épisodes

  1. Overcoming a Delivery Challenge of Retinal Gene Therapies

    Publié: 19/06/2025

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

    Publié: 12/06/2025

  3. How One Patient Organization Drives Drug Development

    Publié: 05/06/2025

  4. Using CRISPR to Modulate Gene Expression

    Publié: 29/05/2025

  5. Satisfying the Hunger for a Prader-Willi Therapy

    Publié: 22/05/2025

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

    Publié: 15/05/2025

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

    Publié: 08/05/2025

  8. An Effort to Build a Better DMD Gene Therapy

    Publié: 02/05/2025

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

    Publié: 24/04/2025

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

    Publié: 17/04/2025

  11. Learning to Take His Vitamins

    Publié: 10/04/2025

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

    Publié: 03/04/2025

  13. Engineering Skin Bacteria to Be Live Biotherapeutics

    Publié: 27/03/2025

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

    Publié: 20/03/2025

  15. My Mother, Myself, and ALS

    Publié: 13/03/2025

  16. From Immovable Object to Advocacy Force

    Publié: 06/03/2025

  17. A Rapid and Scalable Approach for Screening Personalized ASOs

    Publié: 27/02/2025

  18. Addressing the Disease Mechanism of a Rare Kidney Disease

    Publié: 20/02/2025

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

    Publié: 13/02/2025

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

    Publié: 06/02/2025

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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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