RARECast

548 Afleveringen

1. Leveraging Technology to Empower Patients and Decentralize Clinical Trials

   Gepubliceerd: 20-10-2022
2. Equipping The Next-Generation Rare Disease Patient Advocate

   Gepubliceerd: 13-10-2022
3. How a Family Raced from Diagnosis to Experimental Gene Therapy in Three Years

   Gepubliceerd: 6-10-2022
4. How One Advocate Went from Losing His Hearing to Being Heard

   Gepubliceerd: 29-9-2022
5. Building a Pipeline of Therapies to Treat Rare Mineralization Disorders

   Gepubliceerd: 22-9-2022
6. Advancing an Oral Alternative to Infused and Injected Therapies for HAE

   Gepubliceerd: 15-9-2022
7. Embracing the Promise of Patient-Centered Biotechnology

   Gepubliceerd: 8-9-2022
8. Learning to Live with Loss

   Gepubliceerd: 1-9-2022
9. Using Cryptocurrency to Tap into the Wisdom of Crowds

   Gepubliceerd: 25-8-2022
10. A Therapy for a Rare Neurodegenerative Disease Moves Towards Regulatory Review

    Gepubliceerd: 18-8-2022
11. Transforming the Treatment of Neuromuscular Diseases with Next-Gen Oligonucleotides

    Gepubliceerd: 11-8-2022
12. Teaching the Immune System to Let Medicines Do Their Job

    Gepubliceerd: 4-8-2022
13. Designing Clinical Trials with the Patient in Mind

    Gepubliceerd: 28-7-2022
14. Keeping Clinical Trials Running Smoothly

    Gepubliceerd: 21-7-2022
15. A Vision for Patient-Centric Gene Therapy Development

    Gepubliceerd: 14-7-2022
16. Using Genetic Testing to Address Disparities in Care for Kidney Disease

    Gepubliceerd: 7-7-2022
17. Why Reforms to the Accelerated Approval Pathway Threaten Rare Disease Drug Development

    Gepubliceerd: 30-6-2022
18. A Longstanding Academic-Nonprofit Collaboration Gives Rise to an ALS Drug Company

    Gepubliceerd: 16-6-2022
19. Targeting Regulatory RNA to Upregulate Gene Expression to Treat Rare Diseases

    Gepubliceerd: 9-6-2022
20. Developing a New Approach to Treat Rare, Autoimmune Conditions

    Gepubliceerd: 2-6-2022