RARECast

512 Episodes

1. How One Woman Climbed from Patient to Advocate

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

    Published: 6/27/2024
17. Taking a Different Approach to Rare Epilepsies

    Published: 6/20/2024
18. A Rare Disease Drug Developer Tries to Earn Its Stripes

    Published: 6/13/2024
19. Using Plasma to Treat Rare Diseases

    Published: 6/6/2024
20. Differentiating Gene Therapies through Regulatory Elements

    Published: 5/30/2024