RARECast

548 Odcinki

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

   Opublikowany: 30.01.2025
2. Targeting Rare Liver Diseases with Gene Editing Therapies

   Opublikowany: 23.01.2025
3. Using AI to De-Risk Rare Disease Drug Development

   Opublikowany: 16.01.2025
4. Addressing a Growing Demand for Plasma-Derived Therapies

   Opublikowany: 9.01.2025
5. Empowering Patients with Data to Drive Drug Development

   Opublikowany: 2.01.2025
6. How Advocates Are Advancing a Treatment for an Ultra-Rare Disease

   Opublikowany: 26.12.2024
7. Engineering B Cells to Produce Therapeutic Proteins

   Opublikowany: 19.12.2024
8. A Pipeline Win as BridgeBio Learns to Balance Theory with Practice

   Opublikowany: 12.12.2024
9. Considering What It Will Take for Payers to Embrace Advances in Sequencing

   Opublikowany: 5.12.2024
10. Finding Strength

    Opublikowany: 27.11.2024
11. A Non-Profit Works to Advance a DMD Gene Therapy

    Opublikowany: 21.11.2024
12. Accepting What We Can’t Understand

    Opublikowany: 14.11.2024
13. A Gene Therapy Company Born from a Father’s Efforts to Save his Daughter

    Opublikowany: 7.11.2024
14. Lowering Barriers to Clinical Trial Participation with Do-It-Yourself Blood Sample Collection

    Opublikowany: 31.10.2024
15. Scaling N-of-1 Therapies to Viability

    Opublikowany: 24.10.2024
16. Building Gene Therapies that Address Gain and Loss of Function Simultaneously

    Opublikowany: 17.10.2024
17. How One Woman Climbed from Patient to Advocate

    Opublikowany: 10.10.2024
18. Shortening the Diagnostic Odyssey

    Opublikowany: 3.10.2024
19. Making Exomes More Revealing

    Opublikowany: 26.09.2024
20. How One Patient Organization Catalyzed Drug Development

    Opublikowany: 19.09.2024