RARECast

512 Folgen

1. The Psychological Toll Rare Diseases Can Have on Healthy Siblings

   Vom: 29.5.2019
2. Blood Test Can Provide Earlier Diagnosis of Autism Spectrum Disorder

   Vom: 22.5.2019
3. Understanding the Role of Natural History Studies in Clinical Trials

   Vom: 15.5.2019
4. How Two Mothers Discovered That a Drug for One Rare Disorder Might Benefit Another

   Vom: 8.5.2019
5. Shortening the Diagnostic Odyssey

   Vom: 1.5.2019
6. Eliminating Barriers to Adoption of Children with Rare Diseases

   Vom: 24.4.2019
7. A Young Rare Disease Advocate Discusses Life with an Undiagnosed Condition

   Vom: 17.4.2019
8. The Benefits of an Open-Source Registry for Rare Diseases

   Vom: 10.4.2019
9. How a Patient Group Helped Drive Drug Development in Rett Syndrome

   Vom: 3.4.2019
10. How Misperceptions Can Create Barriers to Care

    Vom: 27.3.2019
11. Targeting Repeat Expansion Disorders with Next-Gen Antisense Drugs

    Vom: 21.3.2019
12. Getting Misfolding Proteins to Shape Up

    Vom: 13.3.2019
13. A Different Approach to Inhibiting the Complement System

    Vom: 6.3.2019
14. Looking Back in Time to Find Rare Disease Patients Today

    Vom: 27.2.2019
15. Ionis CEO Stanley Crooke Discusses Success as a Platform Technology Company

    Vom: 20.2.2019
16. Orchard Looks toward Harvesting Expanded Gene Therapy Pipeline

    Vom: 13.2.2019
17. Connecting Rare Disease Patients in India and the United States

    Vom: 6.2.2019
18. Regenerative Medicine Moves into the Spotlight

    Vom: 30.1.2019
19. Learning New Tricks from Pathogenic Bacteria to Target the Microbiome

    Vom: 24.1.2019
20. Orphan Drug Accelerator Spin-Out Tackles Rare Endocrine Disorders

    Vom: 16.1.2019