RARECast

548 Episodes

1. Addressing the Delivery Challenges of Genetic Medicines

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

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

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

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

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

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

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

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

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

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

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

    Published: 6/3/2021
13. A New Therapy Offers A Different Approach to Inhibiting the Complement System

    Published: 5/28/2021
14. Bringing Aberrant Proteins Back into the Fold

    Published: 5/20/2021
15. A Cat who Curates Rare Mice

    Published: 5/13/2021
16. Putting the Patient at the Center of Rare Disease Clinical Trials

    Published: 5/6/2021
17. Creating a Toolkit to Accelerate the Development of Gene Editing Therapies

    Published: 4/29/2021
18. PTC Looks to Advanced Therapies

    Published: 4/22/2021
19. Going to Extreme Lengths

    Published: 4/15/2021
20. Everything’s up to Date in Kansas City, at Least When It Comes to Genomics

    Published: 4/8/2021