RARECast

548 Episodes

1. Leveraging Technology to Empower Patients and Decentralize Clinical Trials

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

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

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

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

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

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

   Published: 9/8/2022
8. Learning to Live with Loss

   Published: 9/1/2022
9. Using Cryptocurrency to Tap into the Wisdom of Crowds

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

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

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

    Published: 8/4/2022
13. Designing Clinical Trials with the Patient in Mind

    Published: 7/28/2022
14. Keeping Clinical Trials Running Smoothly

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

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

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

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

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

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

    Published: 6/2/2022