RARECast

516 Episodes

1. Targeting the Leading Cause of Death in Friedreich’s Ataxia with a Gene Therapy

   Published: 2/1/2024
2. How Inhaled mRNA May Help Rare Disease Patients Breathe Easier

   Published: 1/25/2024
3. Changing What’s Possible with Cell and Gene Therapies

   Published: 1/18/2024
4. Why Flying with a Wheelchair Is a Civil Rights Issue

   Published: 1/11/2024
5. Mapping Spheres of Influence

   Published: 1/4/2024
6. Bringing Precision to the Treatment of Rare Cancers

   Published: 12/28/2023
7. The Challenges of Developing a Therapy for Pregnant Women with A Rare Condition

   Published: 12/21/2023
8. How One Drug Developer Is Addressing Health Inequities

   Published: 12/14/2023
9. Accelerating the Development of Genetic Medicines with AI

   Published: 12/7/2023
10. Leveraging AI to Address the Neuropsychiatric Conditions Associated with a Rare Disease

    Published: 11/30/2023
11. In Pursuit of a Treatment for His Son, A Lawyer Becomes a Biotech Executive

    Published: 11/22/2023
12. In Push for Treatment, a Patient Organization Becomes a Trial Sponsor

    Published: 11/16/2023
13. Delayed Diagnosis of Rare Diseases Takes Staggering Economic Toll

    Published: 11/9/2023
14. A Cell Therapy to Control Heart-Damaging Inflammation in Duchenne

    Published: 11/2/2023
15. Stimulating the Growth of Muscle Mass in People with Neuromuscular Diseases

    Published: 10/26/2023
16. Turning Grief into Action

    Published: 10/19/2023
17. A Plea to the FDA to Give an Ultra-Rare Disease Drug a Fair Hearing

    Published: 10/12/2023
18. How the Woolly Mammoth Beat a Path to Better Gene Therapies

    Published: 10/5/2023
19. A Genetic Counselor’s Journey into Patient Advocacy

    Published: 9/28/2023
20. A Company in the Fold of Rare Disease

    Published: 9/21/2023