RARECast

512 Episodes

1. Aspa Therapeutics Forges Gene Therapy for Rare Neurological Condition

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

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

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

   Published: 12/19/2018
5. Late-Stage Trial Underway for Treatment of Rare Obesity Disorder

   Published: 12/12/2018
6. RegenxBio Bets Its Gene Therapy Technology Provides Competitive Edge

   Published: 12/5/2018
7. The Potential for Digital Health Technologies to Transform Clinical Trials

   Published: 11/28/2018
8. Matching Volunteers to Rare Disease Organizations in Need

   Published: 11/21/2018
9. How One Rare Disease Organization Used Technology to Provide Greater Patient Insight to FDA

   Published: 11/14/2018
10. The Potentially Deadly Consequences of an Oversight in Newborn Screening Tests

    Published: 11/7/2018
11. Advancing a New Approach to Sickle Cell Disease

    Published: 10/31/2018
12. Conquering Life with a Rare Disease

    Published: 10/24/2018
13. Why Rare Disease Drug Developers Need Regular Interaction with the FDA

    Published: 10/17/2018
14. Making the Case for an FDA Rare Disease Center of Excellence

    Published: 10/10/2018
15. An Elite Runner Makes a Cross-Canada Trek for His Son and Rare Disease

    Published: 10/3/2018
16. How Rare Disease Advocates Can Get The Most out of Collaborations

    Published: 9/26/2018
17. Establishing Clinical Standards for a Rare Disease

    Published: 9/19/2018
18. Teen Rare Disease Advocate Fights the Condition That Took His Brother's Life

    Published: 9/12/2018
19. Addressing the Emotional Toll of Rare Diseases

    Published: 9/5/2018
20. Forging a Research Agenda for a Rare Disease

    Published: 8/29/2018