RARECast

516 Episodes

1. Forging a New Path for Medicine

   Published: 4/27/2023
2. Restoring Hearing through Gene Therapies

   Published: 4/20/2023
3. Creating a Sustainable Reimbursement Model for Ultra-Rare Therapies

   Published: 4/13/2023
4. Lumos Hopes Its Oral Alternative to HGH Will Fuel Its Growth

   Published: 4/5/2023
5. Advancing a Gene Therapy for a Rare and Fatal CNS Disorder

   Published: 3/30/2023
6. Addressing the Barriers to Patient Participation in Clinical Trials

   Published: 3/23/2023
7. A Venture Philanthropist Makes the Case for Advocates Taking Equity

   Published: 3/16/2023
8. Seeking a Sustainable Business Developing on N-of-1 Therapies

   Published: 3/9/2023
9. How One Drugmaker Reaches out to Communities at Greater Risk for Rare Heart Condition

   Published: 3/3/2023
10. A Next-Generation RNA Therapy Targets Telomere Disorders

    Published: 3/3/2023
11. BridgeBio Advances Therapy for Limb-Girdle Muscular Dystrophy that Started with Two Patient Families

    Published: 2/24/2023
12. Translating Urgency into Science

    Published: 2/16/2023
13. Empowering Ultra-Rare Disease Patients to Pursue the Discovery of Treatments

    Published: 2/10/2023
14. Why a Topical Cannabidiol Gel May Help Treat the Behavioral Symptoms of Fragile X

    Published: 2/2/2023
15. An Ultra-Rare Disease Drug Developer Tries to Navigate Regulatory Uncertainty

    Published: 1/26/2023
16. Powering Cells in People with Rare Mitochondrial Diseases

    Published: 1/19/2023
17. Powering Weakened and Stressed Cells in ALS to Function Better with Nanocrystal Therapy

    Published: 1/12/2023
18. Developing a New Class of Therapies Based on a Natural Cargo Carrier

    Published: 1/5/2023
19. Reaching Beyond the Limits of Enzyme Replacement Therapies with Gene Therapies

    Published: 12/29/2022
20. One Woman’s Journey as a Caregiver to a Husband with Frontotemporal Dementia

    Published: 12/22/2022