RARECast

548 Episodes

1. Deriving Rare Disease Therapies from Human Plasma

   Published: 10/18/2017
2. Learning to Live with Purpose

   Published: 10/11/2017
3. A Mother's Race to Find a Treatment for Her Daughter's Ultra-Rare Condition

   Published: 10/4/2017
4. Screening for Rare Diseases

   Published: 9/27/2017
5. A Look at the Changing World of Work for People with a Chronic Illness

   Published: 9/20/2017
6. Aligning Interests in Rare Disease Partnerships

   Published: 9/13/2017
7. A Veteran Venture Capitalist Discusses Investing in Rare Disease Drug Development

   Published: 9/6/2017
8. A Patient Group Crafts Guidelines for Working with Pharma

   Published: 8/30/2017
9. Bringing Innovation to Business Models for Rare Disease Drug Development

   Published: 8/23/2017
10. Rethinking Accessibility and Dependency

    Published: 8/16/2017
11. How Simon Wheatcroft, Blinded by a Rare Disease, Became an Ultramarathoner

    Published: 8/9/2017
12. Spark Hoping Its Luxturna Will Become First FDA-Approved Gene Therapy

    Published: 8/2/2017
13. What Rare Patient-Investors Can Learn From Venture Capitalists

    Published: 7/26/2017
14. Novel Effort Delivers Failed DMD Drug to Former Clinical Trial Participants

    Published: 7/19/2017
15. Centogene Seeks to Deliver Diagnoses to Rare Disease Patients Early

    Published: 7/12/2017
16. Albireo Advances Pipeline for Rare Liver and Gastrointestinal Diseases

    Published: 7/5/2017
17. aTyr Pharma Seeks to Modulate Activity in FSHD and Other Rare Disease

    Published: 6/28/2017
18. Summit Pursues A Different Approach to Treating Duchenne

    Published: 6/21/2017
19. Delivering Enzyme Replacement Therapies Across the Blood-Brain Barrier

    Published: 6/14/2017
20. Sangamo Advances Gene Editing Therapies for Multiple Rare Diseases into the Clinic

    Published: 6/7/2017