RARECast

548 Episoder

1. Shortening the Diagnostic Odyssey

   Publisert: 1.5.2019
2. Eliminating Barriers to Adoption of Children with Rare Diseases

   Publisert: 24.4.2019
3. A Young Rare Disease Advocate Discusses Life with an Undiagnosed Condition

   Publisert: 17.4.2019
4. The Benefits of an Open-Source Registry for Rare Diseases

   Publisert: 10.4.2019
5. How a Patient Group Helped Drive Drug Development in Rett Syndrome

   Publisert: 3.4.2019
6. How Misperceptions Can Create Barriers to Care

   Publisert: 27.3.2019
7. Targeting Repeat Expansion Disorders with Next-Gen Antisense Drugs

   Publisert: 21.3.2019
8. Getting Misfolding Proteins to Shape Up

   Publisert: 13.3.2019
9. A Different Approach to Inhibiting the Complement System

   Publisert: 6.3.2019
10. Looking Back in Time to Find Rare Disease Patients Today

    Publisert: 27.2.2019
11. Ionis CEO Stanley Crooke Discusses Success as a Platform Technology Company

    Publisert: 20.2.2019
12. Orchard Looks toward Harvesting Expanded Gene Therapy Pipeline

    Publisert: 13.2.2019
13. Connecting Rare Disease Patients in India and the United States

    Publisert: 6.2.2019
14. Regenerative Medicine Moves into the Spotlight

    Publisert: 30.1.2019
15. Learning New Tricks from Pathogenic Bacteria to Target the Microbiome

    Publisert: 24.1.2019
16. Orphan Drug Accelerator Spin-Out Tackles Rare Endocrine Disorders

    Publisert: 16.1.2019
17. Aspa Therapeutics Forges Gene Therapy for Rare Neurological Condition

    Publisert: 9.1.2019
18. How Patient-Led Collaborations Are Transforming Rare Disease Drug Discovery and Development

    Publisert: 2.1.2019
19. Sobi Expands Its Immunology Franchise with HLH Drug Approval

    Publisert: 27.12.2018
20. Learning to Thrive

    Publisert: 19.12.2018