RARECast

548 Episodio

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

   Pubblicato: 03/03/2023
2. A Next-Generation RNA Therapy Targets Telomere Disorders

   Pubblicato: 03/03/2023
3. BridgeBio Advances Therapy for Limb-Girdle Muscular Dystrophy that Started with Two Patient Families

   Pubblicato: 24/02/2023
4. Translating Urgency into Science

   Pubblicato: 16/02/2023
5. Empowering Ultra-Rare Disease Patients to Pursue the Discovery of Treatments

   Pubblicato: 10/02/2023
6. Why a Topical Cannabidiol Gel May Help Treat the Behavioral Symptoms of Fragile X

   Pubblicato: 02/02/2023
7. An Ultra-Rare Disease Drug Developer Tries to Navigate Regulatory Uncertainty

   Pubblicato: 26/01/2023
8. Powering Cells in People with Rare Mitochondrial Diseases

   Pubblicato: 19/01/2023
9. Powering Weakened and Stressed Cells in ALS to Function Better with Nanocrystal Therapy

   Pubblicato: 12/01/2023
10. Developing a New Class of Therapies Based on a Natural Cargo Carrier

    Pubblicato: 05/01/2023
11. Reaching Beyond the Limits of Enzyme Replacement Therapies with Gene Therapies

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

    Pubblicato: 22/12/2022
13. Treating Rare Endocrine Disorders with Therapeutic Peptides

    Pubblicato: 15/12/2022
14. Tackling the Pricing Challenges for Advanced Therapies for Rare Diseases

    Pubblicato: 08/12/2022
15. Addressing the Current Limitations of AAV Gene Therapies

    Pubblicato: 01/12/2022
16. Examining the Legislative Landscape for Rare Disease Drug Development

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

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

    Pubblicato: 10/11/2022
19. Addressing Racial Disparities in a Rare Blood Cancer

    Pubblicato: 03/11/2022
20. How New Sequencing Technology Promises to Alter the Diagnostic Odyssey

    Pubblicato: 27/10/2022