RARECast

516 Episodes

1. A Gene Therapy Company Born from a Father’s Efforts to Save his Daughter

   Published: 11/7/2024
2. Lowering Barriers to Clinical Trial Participation with Do-It-Yourself Blood Sample Collection

   Published: 10/31/2024
3. Scaling N-of-1 Therapies to Viability

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

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

   Published: 10/10/2024
6. Shortening the Diagnostic Odyssey

   Published: 10/3/2024
7. Making Exomes More Revealing

   Published: 9/26/2024
8. How One Patient Organization Catalyzed Drug Development

   Published: 9/19/2024
9. Enabling On-Demand Therapies

   Published: 9/16/2024
10. After Driving an N-of-1 Therapy for Her Son, an Advocate Turns to Helping Others

    Published: 9/5/2024
11. Putting Rare Disease Organization in the Business of Drug Development

    Published: 8/29/2024
12. Understanding the Many Questions Gene Therapies Raise

    Published: 8/22/2024
13. A Test to Identify People Likely to Develop ALS

    Published: 8/15/2024
14. How an Ultra-Rare Disease Patient Foundation Advanced a Gene Therapy

    Published: 8/8/2024
15. Making the $10 Genome a Reality

    Published: 8/1/2024
16. A No-Nonsense Effort to Develop a Therapy that Works Across Genetic Diseases

    Published: 7/25/2024
17. Mapping a Perilous Journey with Humor

    Published: 7/18/2024
18. Restoring Vision in Inherited Retinal Disease

    Published: 7/11/2024
19. Bringing Genome Sequencing to Rural Populations

    Published: 7/4/2024
20. Addressing a Blind Spot in Treatments for the Cornea

    Published: 6/27/2024