RARECast

548 Avsnitt

1. Overcoming a Delivery Challenge of Retinal Gene Therapies

   Publicerades: 2025-06-19
2. A Gene Editing First Augurs an Era of Bespoke Therapies

   Publicerades: 2025-06-12
3. How One Patient Organization Drives Drug Development

   Publicerades: 2025-06-05
4. Using CRISPR to Modulate Gene Expression

   Publicerades: 2025-05-29
5. Satisfying the Hunger for a Prader-Willi Therapy

   Publicerades: 2025-05-22
6. How an Academic Medical Center Helped Change the Landscape for a Rare Disease

   Publicerades: 2025-05-15
7. Improving Outcomes for People with a Set of Rare Cancers

   Publicerades: 2025-05-08
8. An Effort to Build a Better DMD Gene Therapy

   Publicerades: 2025-05-02
9. A Once-Failed Pain Therapy Shows Potential in a Neurodevelopmental Disorder

   Publicerades: 2025-04-24
10. Advancing a Cutting-Edge Therapy for a Rare, Childhood Cancer

    Publicerades: 2025-04-17
11. Learning to Take His Vitamins

    Publicerades: 2025-04-10
12. The First Treatment for a Rare Neurodegenerative Condition Awaits FDA Approval

    Publicerades: 2025-04-03
13. Engineering Skin Bacteria to Be Live Biotherapeutics

    Publicerades: 2025-03-27
14. Seeing the Gene and Cell Therapy Translational Divide as an Opportunity

    Publicerades: 2025-03-20
15. My Mother, Myself, and ALS

    Publicerades: 2025-03-13
16. From Immovable Object to Advocacy Force

    Publicerades: 2025-03-06
17. A Rapid and Scalable Approach for Screening Personalized ASOs

    Publicerades: 2025-02-27
18. Addressing the Disease Mechanism of a Rare Kidney Disease

    Publicerades: 2025-02-20
19. The Mother of a Son with SCD, Applies Direct Experience to Her Clinical Trials Work

    Publicerades: 2025-02-13
20. A First for Rett Syndrome with More in the Pipeline

    Publicerades: 2025-02-06