RARECast

548 Odcinki

1. Overcoming a Delivery Challenge of Retinal Gene Therapies

   Opublikowany: 19.06.2025
2. A Gene Editing First Augurs an Era of Bespoke Therapies

   Opublikowany: 12.06.2025
3. How One Patient Organization Drives Drug Development

   Opublikowany: 5.06.2025
4. Using CRISPR to Modulate Gene Expression

   Opublikowany: 29.05.2025
5. Satisfying the Hunger for a Prader-Willi Therapy

   Opublikowany: 22.05.2025
6. How an Academic Medical Center Helped Change the Landscape for a Rare Disease

   Opublikowany: 15.05.2025
7. Improving Outcomes for People with a Set of Rare Cancers

   Opublikowany: 8.05.2025
8. An Effort to Build a Better DMD Gene Therapy

   Opublikowany: 2.05.2025
9. A Once-Failed Pain Therapy Shows Potential in a Neurodevelopmental Disorder

   Opublikowany: 24.04.2025
10. Advancing a Cutting-Edge Therapy for a Rare, Childhood Cancer

    Opublikowany: 17.04.2025
11. Learning to Take His Vitamins

    Opublikowany: 10.04.2025
12. The First Treatment for a Rare Neurodegenerative Condition Awaits FDA Approval

    Opublikowany: 3.04.2025
13. Engineering Skin Bacteria to Be Live Biotherapeutics

    Opublikowany: 27.03.2025
14. Seeing the Gene and Cell Therapy Translational Divide as an Opportunity

    Opublikowany: 20.03.2025
15. My Mother, Myself, and ALS

    Opublikowany: 13.03.2025
16. From Immovable Object to Advocacy Force

    Opublikowany: 6.03.2025
17. A Rapid and Scalable Approach for Screening Personalized ASOs

    Opublikowany: 27.02.2025
18. Addressing the Disease Mechanism of a Rare Kidney Disease

    Opublikowany: 20.02.2025
19. The Mother of a Son with SCD, Applies Direct Experience to Her Clinical Trials Work

    Opublikowany: 13.02.2025
20. A First for Rett Syndrome with More in the Pipeline

    Opublikowany: 6.02.2025