RARECast

548 Episodes

1. Addressing a Shortage of Genetic Counselors with AI

   Published: 4/25/2024
2. A Small Molecule Therapy to Regenerate Muscle in People with DMD

   Published: 4/18/2024
3. Forging a Faster Path for Gene Therapies

   Published: 4/11/2024
4. Determining the Value of Rare Disease Therapies

   Published: 4/4/2024
5. Buying and Building a Gene Therapy Presence

   Published: 3/28/2024
6. A Clinical Trial Failure Derails a Promising Technology

   Published: 3/21/2024
7. Helping People with Undiagnosed Rare Diseases Find Answers

   Published: 3/14/2024
8. Speeding and Scaling the Development of Genome Editing Therapies

   Published: 3/7/2024
9. Getting a Next-Generation Genome Editing Therapy for Sickle Cell Disease Back on Track

   Published: 2/29/2024
10. Trying to Break a Leg in a Wheelchair

    Published: 2/22/2024
11. A Drug Developer that Makes Pediatric Cancers a Priority

    Published: 2/15/2024
12. Harnessing the Body’s Natural RNA Machinery to Treat Diseases

    Published: 2/8/2024
13. Targeting the Leading Cause of Death in Friedreich’s Ataxia with a Gene Therapy

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

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

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

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

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

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

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

    Published: 12/14/2023