RARECast

548 Afleveringen

1. How One Drugmaker Reaches out to Communities at Greater Risk for Rare Heart Condition

   Gepubliceerd: 3-3-2023
2. A Next-Generation RNA Therapy Targets Telomere Disorders

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

   Gepubliceerd: 24-2-2023
4. Translating Urgency into Science

   Gepubliceerd: 16-2-2023
5. Empowering Ultra-Rare Disease Patients to Pursue the Discovery of Treatments

   Gepubliceerd: 10-2-2023
6. Why a Topical Cannabidiol Gel May Help Treat the Behavioral Symptoms of Fragile X

   Gepubliceerd: 2-2-2023
7. An Ultra-Rare Disease Drug Developer Tries to Navigate Regulatory Uncertainty

   Gepubliceerd: 26-1-2023
8. Powering Cells in People with Rare Mitochondrial Diseases

   Gepubliceerd: 19-1-2023
9. Powering Weakened and Stressed Cells in ALS to Function Better with Nanocrystal Therapy

   Gepubliceerd: 12-1-2023
10. Developing a New Class of Therapies Based on a Natural Cargo Carrier

    Gepubliceerd: 5-1-2023
11. Reaching Beyond the Limits of Enzyme Replacement Therapies with Gene Therapies

    Gepubliceerd: 29-12-2022
12. One Woman’s Journey as a Caregiver to a Husband with Frontotemporal Dementia

    Gepubliceerd: 22-12-2022
13. Treating Rare Endocrine Disorders with Therapeutic Peptides

    Gepubliceerd: 15-12-2022
14. Tackling the Pricing Challenges for Advanced Therapies for Rare Diseases

    Gepubliceerd: 8-12-2022
15. Addressing the Current Limitations of AAV Gene Therapies

    Gepubliceerd: 1-12-2022
16. Examining the Legislative Landscape for Rare Disease Drug Development

    Gepubliceerd: 24-11-2022
17. How a Drug Setback Became a Patient Community’s Gain

    Gepubliceerd: 18-11-2022
18. Helping Regulators and Drug Developers Understand the Challenges of Living with Fabry Disease

    Gepubliceerd: 10-11-2022
19. Addressing Racial Disparities in a Rare Blood Cancer

    Gepubliceerd: 3-11-2022
20. How New Sequencing Technology Promises to Alter the Diagnostic Odyssey

    Gepubliceerd: 27-10-2022