RARECast

512 Episodes

1. The Decision to Get Tested for Huntington's Disease and How It Changed Seth Rotberg's Life

   Published: 4/4/2018
2. Nonprofit Sees Need in Developing Gene Therapies for Inherited Eye Disesases

   Published: 3/29/2018
3. Casting a Wider Net for Genetic Diseases

   Published: 3/21/2018
4. Corbus Seeks to Quiet the Immune System in Chronic Conditions

   Published: 3/14/2018
5. Addressing the Challenges of Rare Disease Drug Development

   Published: 3/7/2018
6. Giving Voice to Patients at the FDA

   Published: 2/28/2018
7. Finding a Rare Friend

   Published: 2/21/2018
8. Why a Sports Physical May Trigger a Doctor to Think About a Rare Diagnosis

   Published: 2/14/2018
9. UPenn's Orphan Disease Center Takes a Rare Approach

   Published: 2/7/2018
10. How Patients Can Accelerate Translational Research in Rare Disease

    Published: 1/31/2018
11. Notre Dame Center Leverages Commonalities of Rare and Neglected Diseases

    Published: 1/24/2018
12. GalXC Quest: Dicerna Bets on Its Unqiue Approach to RNAi

    Published: 1/17/2018
13. Pharnext Takes a Unique Twist on Repurposing Drugs for Rare Diseases

    Published: 1/10/2018
14. uniQure Seeks to Find Elusive Market Success with Hemophilia B Gene Therapy

    Published: 1/3/2018
15. Savara Advances Pipeline of Inhaled Therapeutics for Rare Lung Diseases

    Published: 12/27/2017
16. Why the Microbiome May Provide a Novel Approach to Treating Certain Rare Diseases

    Published: 12/20/2017
17. FDA Outlines a Collaborative Approach for Pediatric Rare Disease Trials

    Published: 12/13/2017
18. Why Patient-Centred Outcomes Are Critical to Rare Disease Drug Trials

    Published: 12/6/2017
19. What the FDA's New Framework Means for Regenerative Therapies

    Published: 11/29/2017
20. Measuring the Value of Therapies for Ultra-Rare Diseases

    Published: 11/22/2017