RARECast

548 Episodio

1. Overcoming a Delivery Challenge of Retinal Gene Therapies

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

   Pubblicato: 12/06/2025
3. How One Patient Organization Drives Drug Development

   Pubblicato: 05/06/2025
4. Using CRISPR to Modulate Gene Expression

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

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

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

   Pubblicato: 08/05/2025
8. An Effort to Build a Better DMD Gene Therapy

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

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

    Pubblicato: 17/04/2025
11. Learning to Take His Vitamins

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

    Pubblicato: 03/04/2025
13. Engineering Skin Bacteria to Be Live Biotherapeutics

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

    Pubblicato: 20/03/2025
15. My Mother, Myself, and ALS

    Pubblicato: 13/03/2025
16. From Immovable Object to Advocacy Force

    Pubblicato: 06/03/2025
17. A Rapid and Scalable Approach for Screening Personalized ASOs

    Pubblicato: 27/02/2025
18. Addressing the Disease Mechanism of a Rare Kidney Disease

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

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

    Pubblicato: 06/02/2025