RARECast

512 Episodes

1. How a Drug Setback Became a Patient Community’s Gain

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

   Published: 11/10/2022
3. Addressing Racial Disparities in a Rare Blood Cancer

   Published: 11/3/2022
4. How New Sequencing Technology Promises to Alter the Diagnostic Odyssey

   Published: 10/27/2022
5. Leveraging Technology to Empower Patients and Decentralize Clinical Trials

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

    Published: 7/7/2022