RARECast

516 Episodes

1. Marrying Antibodies and Oligonucleotides to Target Genetic Medicine

   Published: 6/26/2019
2. What Rare Disease Patients Should Understand about Genetics

   Published: 6/19/2019
3. Program Seeks to Drive Collaborative Research Model Among Rare Disease Groups

   Published: 6/12/2019
4. A Gene Hunter Becomes a Drug Hunter Too

   Published: 6/5/2019
5. The Psychological Toll Rare Diseases Can Have on Healthy Siblings

   Published: 5/29/2019
6. Blood Test Can Provide Earlier Diagnosis of Autism Spectrum Disorder

   Published: 5/22/2019
7. Understanding the Role of Natural History Studies in Clinical Trials

   Published: 5/15/2019
8. How Two Mothers Discovered That a Drug for One Rare Disorder Might Benefit Another

   Published: 5/8/2019
9. Shortening the Diagnostic Odyssey

   Published: 5/1/2019
10. Eliminating Barriers to Adoption of Children with Rare Diseases

    Published: 4/24/2019
11. A Young Rare Disease Advocate Discusses Life with an Undiagnosed Condition

    Published: 4/17/2019
12. The Benefits of an Open-Source Registry for Rare Diseases

    Published: 4/10/2019
13. How a Patient Group Helped Drive Drug Development in Rett Syndrome

    Published: 4/3/2019
14. How Misperceptions Can Create Barriers to Care

    Published: 3/27/2019
15. Targeting Repeat Expansion Disorders with Next-Gen Antisense Drugs

    Published: 3/21/2019
16. Getting Misfolding Proteins to Shape Up

    Published: 3/13/2019
17. A Different Approach to Inhibiting the Complement System

    Published: 3/6/2019
18. Looking Back in Time to Find Rare Disease Patients Today

    Published: 2/27/2019
19. Ionis CEO Stanley Crooke Discusses Success as a Platform Technology Company

    Published: 2/20/2019
20. Orchard Looks toward Harvesting Expanded Gene Therapy Pipeline

    Published: 2/13/2019