RARECast

532 Odcinki

1. A Rapid and Scalable Approach for Screening Personalized ASOs

   Opublikowany: 27.02.2025
2. Addressing the Disease Mechanism of a Rare Kidney Disease

   Opublikowany: 20.02.2025
3. The Mother of a Son with SCD, Applies Direct Experience to Her Clinical Trials Work

   Opublikowany: 13.02.2025
4. A First for Rett Syndrome with More in the Pipeline

   Opublikowany: 6.02.2025
5. A Heart Attack that Led to a Rare Disease Diagnosis

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

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

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

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

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

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

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

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

    Opublikowany: 5.12.2024
14. Finding Strength

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

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

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

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

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

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

    Opublikowany: 17.10.2024