RARECast

512 Folgen

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

   Vom: 30.3.2023
2. Addressing the Barriers to Patient Participation in Clinical Trials

   Vom: 23.3.2023
3. A Venture Philanthropist Makes the Case for Advocates Taking Equity

   Vom: 16.3.2023
4. Seeking a Sustainable Business Developing on N-of-1 Therapies

   Vom: 9.3.2023
5. How One Drugmaker Reaches out to Communities at Greater Risk for Rare Heart Condition

   Vom: 3.3.2023
6. A Next-Generation RNA Therapy Targets Telomere Disorders

   Vom: 3.3.2023
7. BridgeBio Advances Therapy for Limb-Girdle Muscular Dystrophy that Started with Two Patient Families

   Vom: 24.2.2023
8. Translating Urgency into Science

   Vom: 16.2.2023
9. Empowering Ultra-Rare Disease Patients to Pursue the Discovery of Treatments

   Vom: 10.2.2023
10. Why a Topical Cannabidiol Gel May Help Treat the Behavioral Symptoms of Fragile X

    Vom: 2.2.2023
11. An Ultra-Rare Disease Drug Developer Tries to Navigate Regulatory Uncertainty

    Vom: 26.1.2023
12. Powering Cells in People with Rare Mitochondrial Diseases

    Vom: 19.1.2023
13. Powering Weakened and Stressed Cells in ALS to Function Better with Nanocrystal Therapy

    Vom: 12.1.2023
14. Developing a New Class of Therapies Based on a Natural Cargo Carrier

    Vom: 5.1.2023
15. Reaching Beyond the Limits of Enzyme Replacement Therapies with Gene Therapies

    Vom: 29.12.2022
16. One Woman’s Journey as a Caregiver to a Husband with Frontotemporal Dementia

    Vom: 22.12.2022
17. Treating Rare Endocrine Disorders with Therapeutic Peptides

    Vom: 15.12.2022
18. Tackling the Pricing Challenges for Advanced Therapies for Rare Diseases

    Vom: 8.12.2022
19. Addressing the Current Limitations of AAV Gene Therapies

    Vom: 1.12.2022
20. Examining the Legislative Landscape for Rare Disease Drug Development

    Vom: 24.11.2022