RARECast

512 Folgen

1. How an Ultra-Rare Disease Patient Organization Drove Research to a Treatment

   Vom: 10.12.2020
2. With a New Identity, Travere Thearpeutics Seeks to Find Its True Path

   Vom: 4.12.2020
3. Making Gene Therapies Accessible to Patient with Ultra-Rare Conditions

   Vom: 26.11.2020
4. Using RNA Therapies to Target Inherited Retinal Diseases

   Vom: 20.11.2020
5. Part 4-Gene Therapy: A Researcher’s Perspective

   Vom: 13.11.2020
6. Part 3-Gene Therapy: An Industry Perspective

   Vom: 6.11.2020
7. Part 2-Gene Therapy: A Patient Advocate's Perspective

   Vom: 29.10.2020
8. Part 1-Gene Therapy: A Genetic Counselor’s Perspective

   Vom: 23.10.2020
9. An App to Make Patients the Experts in their Own Conditions

   Vom: 15.10.2020
10. Pursuing a New Target for Autoimmune Diseases

    Vom: 8.10.2020
11. Finding a Rare Friend

    Vom: 2.10.2020
12. Industrializing Individual Therapies for People with Rare Conditions

    Vom: 25.9.2020
13. Rare Disease Advocate Turned Filmmaker Turns Lens on his Son

    Vom: 17.9.2020
14. Inhibiting Signaling that Drives Growth of Rare Malformations

    Vom: 10.9.2020
15. Readying a Rare Disease Community for the Availability of Treatments

    Vom: 3.9.2020
16. Forging a Rare Disease Research Agenda

    Vom: 27.8.2020
17. Delivering Injectable Drugs Orally

    Vom: 20.8.2020
18. What Young Adults with Rare Conditions Need to Know About Health Insurance

    Vom: 14.8.2020
19. Mirum Prepares to Seek Approval for Rare Liver Disease Therapy

    Vom: 7.8.2020
20. Payer Perceptions of Fairness in Pricing Rare Disease Therapies

    Vom: 4.8.2020