RARECast

516 Episodes

1. Treating Rare Endocrine Disorders with Therapeutic Peptides

   Published: 12/15/2022
2. Tackling the Pricing Challenges for Advanced Therapies for Rare Diseases

   Published: 12/8/2022
3. Addressing the Current Limitations of AAV Gene Therapies

   Published: 12/1/2022
4. Examining the Legislative Landscape for Rare Disease Drug Development

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

    Published: 8/4/2022