RARECast

548 Episodes

1. A Heart Attack that Led to a Rare Disease Diagnosis

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

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

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

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

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

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

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

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

   Published: 12/5/2024
10. Finding Strength

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

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

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

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

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

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

    Published: 10/17/2024
17. How One Woman Climbed from Patient to Advocate

    Published: 10/10/2024
18. Shortening the Diagnostic Odyssey

    Published: 10/3/2024
19. Making Exomes More Revealing

    Published: 9/26/2024
20. How One Patient Organization Catalyzed Drug Development

    Published: 9/19/2024