RARECast

516 Episodes

1. What Rare Patient-Investors Can Learn From Venture Capitalists

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

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

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

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

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

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

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

   Published: 6/7/2017
9. Navigating the Challenges of Rare Disease Drug Development

   Published: 5/31/2017
10. Accelerating Rare Disease Drug Discovery

    Published: 5/24/2017
11. Experimental Rare Disease Therapy Wins New FDA Reg Med Designation

    Published: 5/17/2017
12. How Rare Disease Patients Can Get their Voices Heard in Drug Discovery and Development

    Published: 5/10/2017
13. One Patient's Difficult Road to a Rare Diagnosis

    Published: 5/3/2017
14. How Researchers Diagnosed Four Patients with a Never-Before-Identified Rare Disease in a Day

    Published: 4/26/2017
15. Stem Cell Gene Therapy Restores Immune System in Kids with SCID

    Published: 4/19/2017
16. Rare Disease Advocates Push for Incentives to Drugmakers Pulled from Cures Act

    Published: 4/12/2017
17. Non-Profit Drug Company Seeks to Advance Rare Disease Therapies

    Published: 4/5/2017
18. Innovative Collaboration Model Drives Treatment for Rare Disease

    Published: 3/29/2017
19. Girl with Rare Disease Inspires Search for Bone Marrow Donors

    Published: 3/22/2017
20. A Quest for Tribe: Searching for Others with the Same Ultra-Rare Mutation

    Published: 3/15/2017