RARECast

532 Episodes

1. A Rapid and Scalable Approach for Screening Personalized ASOs

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

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

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

   Published: 2/6/2025
5. A Heart Attack that Led to a Rare Disease Diagnosis

   Published: 1/30/2025
6. Targeting Rare Liver Diseases with Gene Editing Therapies

   Published: 1/23/2025
7. Using AI to De-Risk Rare Disease Drug Development

   Published: 1/16/2025
8. Addressing a Growing Demand for Plasma-Derived Therapies

   Published: 1/9/2025
9. Empowering Patients with Data to Drive Drug Development

   Published: 1/2/2025
10. How Advocates Are Advancing a Treatment for an Ultra-Rare Disease

    Published: 12/26/2024
11. Engineering B Cells to Produce Therapeutic Proteins

    Published: 12/19/2024
12. A Pipeline Win as BridgeBio Learns to Balance Theory with Practice

    Published: 12/12/2024
13. Considering What It Will Take for Payers to Embrace Advances in Sequencing

    Published: 12/5/2024
14. Finding Strength

    Published: 11/27/2024
15. A Non-Profit Works to Advance a DMD Gene Therapy

    Published: 11/21/2024
16. Accepting What We Can’t Understand

    Published: 11/14/2024
17. A Gene Therapy Company Born from a Father’s Efforts to Save his Daughter

    Published: 11/7/2024
18. Lowering Barriers to Clinical Trial Participation with Do-It-Yourself Blood Sample Collection

    Published: 10/31/2024
19. Scaling N-of-1 Therapies to Viability

    Published: 10/24/2024
20. Building Gene Therapies that Address Gain and Loss of Function Simultaneously

    Published: 10/17/2024