RARECast

512 Episodes

1. How One Patient Organization Leverages Research Investments

   Published: 5/23/2024
2. Charging into the Storm

   Published: 5/16/2024
3. A Gene Therapy Developer that Embraces Different Models for Reaching Patients

   Published: 5/9/2024
4. Using Directed Evolution to Develop New Vectors for Genetic Medicines

   Published: 5/2/2024
5. Addressing a Shortage of Genetic Counselors with AI

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

    Published: 1/11/2024