RARECast

548 Episodes

1. Overcoming a Delivery Challenge of Retinal Gene Therapies

   Published: 6/19/2025
2. A Gene Editing First Augurs an Era of Bespoke Therapies

   Published: 6/12/2025
3. How One Patient Organization Drives Drug Development

   Published: 6/5/2025
4. Using CRISPR to Modulate Gene Expression

   Published: 5/29/2025
5. Satisfying the Hunger for a Prader-Willi Therapy

   Published: 5/22/2025
6. How an Academic Medical Center Helped Change the Landscape for a Rare Disease

   Published: 5/15/2025
7. Improving Outcomes for People with a Set of Rare Cancers

   Published: 5/8/2025
8. An Effort to Build a Better DMD Gene Therapy

   Published: 5/2/2025
9. A Once-Failed Pain Therapy Shows Potential in a Neurodevelopmental Disorder

   Published: 4/24/2025
10. Advancing a Cutting-Edge Therapy for a Rare, Childhood Cancer

    Published: 4/17/2025
11. Learning to Take His Vitamins

    Published: 4/10/2025
12. The First Treatment for a Rare Neurodegenerative Condition Awaits FDA Approval

    Published: 4/3/2025
13. Engineering Skin Bacteria to Be Live Biotherapeutics

    Published: 3/27/2025
14. Seeing the Gene and Cell Therapy Translational Divide as an Opportunity

    Published: 3/20/2025
15. My Mother, Myself, and ALS

    Published: 3/13/2025
16. From Immovable Object to Advocacy Force

    Published: 3/6/2025
17. A Rapid and Scalable Approach for Screening Personalized ASOs

    Published: 2/27/2025
18. Addressing the Disease Mechanism of a Rare Kidney Disease

    Published: 2/20/2025
19. The Mother of a Son with SCD, Applies Direct Experience to Her Clinical Trials Work

    Published: 2/13/2025
20. A First for Rett Syndrome with More in the Pipeline

    Published: 2/6/2025