RARECast

516 Episodes

1. A Bet on a Unique Set of Vectors

   Published: 10/15/2021
2. How a Rare Diagnosis Impacts a Family

   Published: 10/7/2021
3. Transforming the Rare Disease Landscape with Data

   Published: 10/1/2021
4. Creating a Playbook for Bespoke Gene Therapies

   Published: 9/24/2021
5. Finding Answers for Undiagnosed Patients with Rare Genetic Diseases

   Published: 9/16/2021
6. How a Familiar Face Can Lead to a Rare Disease Diagnosis

   Published: 9/10/2021
7. Bringing the Trial to the Patient

   Published: 9/2/2021
8. Targeting Rare and Chronic Kidney Diseases

   Published: 8/26/2021
9. Addressing the Delivery Challenges of Genetic Medicines

   Published: 8/19/2021
10. Targeting Tissues Throughout the Body with RNA Therapies

    Published: 8/12/2021
11. Delivering Rare Disease Therapies to Patients in Need

    Published: 8/5/2021
12. An Advocate's Journey through Diagnosis, Loss, and Hope

    Published: 7/29/2021
13. Using Model Systems to Find Drugs to Repurpose for Rare Diseases

    Published: 7/22/2021
14. How a Small and Young Foundation Catalyzed Research into a Rare Disease

    Published: 7/16/2021
15. Overcoming the Limitations of Conventional Cell Engineering

    Published: 7/8/2021
16. Experimental Therapy for Rare Endocrine Disorder Offers Hope of Improved Care

    Published: 7/2/2021
17. Forging Gene Therapy Capacity and a Pipeline at the Same Time

    Published: 6/24/2021
18. Using A Natural DNA Repair Process to Improve Genetic Medicines

    Published: 6/18/2021
19. Empowering Rare Disease Patients with their Own Health Records

    Published: 6/10/2021
20. Targeting CNS Disease with Gene Therapies

    Published: 6/3/2021