RARECast

516 Episodes

1. Treating the Root Cause of Sickle Cell Disease

   Published: 1/8/2021
2. Targeting a Common Pathway in Genetic Forms of Obesity

   Published: 12/30/2020
3. After Pruning Orchard, Gaspar Focuses on High Value Opportunities

   Published: 12/23/2020
4. Writing a New Chapter of Genetic Medicine

   Published: 12/17/2020
5. How an Ultra-Rare Disease Patient Organization Drove Research to a Treatment

   Published: 12/10/2020
6. With a New Identity, Travere Thearpeutics Seeks to Find Its True Path

   Published: 12/4/2020
7. Making Gene Therapies Accessible to Patient with Ultra-Rare Conditions

   Published: 11/26/2020
8. Using RNA Therapies to Target Inherited Retinal Diseases

   Published: 11/20/2020
9. Part 4-Gene Therapy: A Researcher’s Perspective

   Published: 11/13/2020
10. Part 3-Gene Therapy: An Industry Perspective

    Published: 11/6/2020
11. Part 2-Gene Therapy: A Patient Advocate's Perspective

    Published: 10/29/2020
12. Part 1-Gene Therapy: A Genetic Counselor’s Perspective

    Published: 10/23/2020
13. An App to Make Patients the Experts in their Own Conditions

    Published: 10/15/2020
14. Pursuing a New Target for Autoimmune Diseases

    Published: 10/8/2020
15. Finding a Rare Friend

    Published: 10/2/2020
16. Industrializing Individual Therapies for People with Rare Conditions

    Published: 9/25/2020
17. Rare Disease Advocate Turned Filmmaker Turns Lens on his Son

    Published: 9/17/2020
18. Inhibiting Signaling that Drives Growth of Rare Malformations

    Published: 9/10/2020
19. Readying a Rare Disease Community for the Availability of Treatments

    Published: 9/3/2020
20. Forging a Rare Disease Research Agenda

    Published: 8/27/2020