RARECast

548 Folgen

1. Overcoming a Delivery Challenge of Retinal Gene Therapies

   Vom: 19.6.2025
2. A Gene Editing First Augurs an Era of Bespoke Therapies

   Vom: 12.6.2025
3. How One Patient Organization Drives Drug Development

   Vom: 5.6.2025
4. Using CRISPR to Modulate Gene Expression

   Vom: 29.5.2025
5. Satisfying the Hunger for a Prader-Willi Therapy

   Vom: 22.5.2025
6. How an Academic Medical Center Helped Change the Landscape for a Rare Disease

   Vom: 15.5.2025
7. Improving Outcomes for People with a Set of Rare Cancers

   Vom: 8.5.2025
8. An Effort to Build a Better DMD Gene Therapy

   Vom: 2.5.2025
9. A Once-Failed Pain Therapy Shows Potential in a Neurodevelopmental Disorder

   Vom: 24.4.2025
10. Advancing a Cutting-Edge Therapy for a Rare, Childhood Cancer

    Vom: 17.4.2025
11. Learning to Take His Vitamins

    Vom: 10.4.2025
12. The First Treatment for a Rare Neurodegenerative Condition Awaits FDA Approval

    Vom: 3.4.2025
13. Engineering Skin Bacteria to Be Live Biotherapeutics

    Vom: 27.3.2025
14. Seeing the Gene and Cell Therapy Translational Divide as an Opportunity

    Vom: 20.3.2025
15. My Mother, Myself, and ALS

    Vom: 13.3.2025
16. From Immovable Object to Advocacy Force

    Vom: 6.3.2025
17. A Rapid and Scalable Approach for Screening Personalized ASOs

    Vom: 27.2.2025
18. Addressing the Disease Mechanism of a Rare Kidney Disease

    Vom: 20.2.2025
19. The Mother of a Son with SCD, Applies Direct Experience to Her Clinical Trials Work

    Vom: 13.2.2025
20. A First for Rett Syndrome with More in the Pipeline

    Vom: 6.2.2025