RARECast

548 Episodio

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

   Pubblicato: 30/01/2025
2. Targeting Rare Liver Diseases with Gene Editing Therapies

   Pubblicato: 23/01/2025
3. Using AI to De-Risk Rare Disease Drug Development

   Pubblicato: 16/01/2025
4. Addressing a Growing Demand for Plasma-Derived Therapies

   Pubblicato: 09/01/2025
5. Empowering Patients with Data to Drive Drug Development

   Pubblicato: 02/01/2025
6. How Advocates Are Advancing a Treatment for an Ultra-Rare Disease

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

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

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

   Pubblicato: 05/12/2024
10. Finding Strength

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

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

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

    Pubblicato: 07/11/2024
14. Lowering Barriers to Clinical Trial Participation with Do-It-Yourself Blood Sample Collection

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

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

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

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

    Pubblicato: 03/10/2024
19. Making Exomes More Revealing

    Pubblicato: 26/09/2024
20. How One Patient Organization Catalyzed Drug Development

    Pubblicato: 19/09/2024