RARECast

548 Episodes

1. Shortening the Diagnostic Odyssey

   Published: 5/1/2019
2. Eliminating Barriers to Adoption of Children with Rare Diseases

   Published: 4/24/2019
3. A Young Rare Disease Advocate Discusses Life with an Undiagnosed Condition

   Published: 4/17/2019
4. The Benefits of an Open-Source Registry for Rare Diseases

   Published: 4/10/2019
5. How a Patient Group Helped Drive Drug Development in Rett Syndrome

   Published: 4/3/2019
6. How Misperceptions Can Create Barriers to Care

   Published: 3/27/2019
7. Targeting Repeat Expansion Disorders with Next-Gen Antisense Drugs

   Published: 3/21/2019
8. Getting Misfolding Proteins to Shape Up

   Published: 3/13/2019
9. A Different Approach to Inhibiting the Complement System

   Published: 3/6/2019
10. Looking Back in Time to Find Rare Disease Patients Today

    Published: 2/27/2019
11. Ionis CEO Stanley Crooke Discusses Success as a Platform Technology Company

    Published: 2/20/2019
12. Orchard Looks toward Harvesting Expanded Gene Therapy Pipeline

    Published: 2/13/2019
13. Connecting Rare Disease Patients in India and the United States

    Published: 2/6/2019
14. Regenerative Medicine Moves into the Spotlight

    Published: 1/30/2019
15. Learning New Tricks from Pathogenic Bacteria to Target the Microbiome

    Published: 1/24/2019
16. Orphan Drug Accelerator Spin-Out Tackles Rare Endocrine Disorders

    Published: 1/16/2019
17. Aspa Therapeutics Forges Gene Therapy for Rare Neurological Condition

    Published: 1/9/2019
18. How Patient-Led Collaborations Are Transforming Rare Disease Drug Discovery and Development

    Published: 1/2/2019
19. Sobi Expands Its Immunology Franchise with HLH Drug Approval

    Published: 12/27/2018
20. Learning to Thrive

    Published: 12/19/2018