RARECast

512 Episodes

1. Advancing a Gene Therapy for a Rare and Fatal CNS Disorder

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

    Published: 12/22/2022
17. Treating Rare Endocrine Disorders with Therapeutic Peptides

    Published: 12/15/2022
18. Tackling the Pricing Challenges for Advanced Therapies for Rare Diseases

    Published: 12/8/2022
19. Addressing the Current Limitations of AAV Gene Therapies

    Published: 12/1/2022
20. Examining the Legislative Landscape for Rare Disease Drug Development

    Published: 11/24/2022