RARECast

548 Episoder

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

   Publisert: 3.3.2023
2. A Next-Generation RNA Therapy Targets Telomere Disorders

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

   Publisert: 24.2.2023
4. Translating Urgency into Science

   Publisert: 16.2.2023
5. Empowering Ultra-Rare Disease Patients to Pursue the Discovery of Treatments

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

   Publisert: 2.2.2023
7. An Ultra-Rare Disease Drug Developer Tries to Navigate Regulatory Uncertainty

   Publisert: 26.1.2023
8. Powering Cells in People with Rare Mitochondrial Diseases

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

   Publisert: 12.1.2023
10. Developing a New Class of Therapies Based on a Natural Cargo Carrier

    Publisert: 5.1.2023
11. Reaching Beyond the Limits of Enzyme Replacement Therapies with Gene Therapies

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

    Publisert: 22.12.2022
13. Treating Rare Endocrine Disorders with Therapeutic Peptides

    Publisert: 15.12.2022
14. Tackling the Pricing Challenges for Advanced Therapies for Rare Diseases

    Publisert: 8.12.2022
15. Addressing the Current Limitations of AAV Gene Therapies

    Publisert: 1.12.2022
16. Examining the Legislative Landscape for Rare Disease Drug Development

    Publisert: 24.11.2022
17. How a Drug Setback Became a Patient Community’s Gain

    Publisert: 18.11.2022
18. Helping Regulators and Drug Developers Understand the Challenges of Living with Fabry Disease

    Publisert: 10.11.2022
19. Addressing Racial Disparities in a Rare Blood Cancer

    Publisert: 3.11.2022
20. How New Sequencing Technology Promises to Alter the Diagnostic Odyssey

    Publisert: 27.10.2022