RARECast

548 Episodo

1. Shortening the Diagnostic Odyssey

   Publicado: 1/5/2019
2. Eliminating Barriers to Adoption of Children with Rare Diseases

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

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

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

   Publicado: 3/4/2019
6. How Misperceptions Can Create Barriers to Care

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

   Publicado: 21/3/2019
8. Getting Misfolding Proteins to Shape Up

   Publicado: 13/3/2019
9. A Different Approach to Inhibiting the Complement System

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

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

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

    Publicado: 13/2/2019
13. Connecting Rare Disease Patients in India and the United States

    Publicado: 6/2/2019
14. Regenerative Medicine Moves into the Spotlight

    Publicado: 30/1/2019
15. Learning New Tricks from Pathogenic Bacteria to Target the Microbiome

    Publicado: 24/1/2019
16. Orphan Drug Accelerator Spin-Out Tackles Rare Endocrine Disorders

    Publicado: 16/1/2019
17. Aspa Therapeutics Forges Gene Therapy for Rare Neurological Condition

    Publicado: 9/1/2019
18. How Patient-Led Collaborations Are Transforming Rare Disease Drug Discovery and Development

    Publicado: 2/1/2019
19. Sobi Expands Its Immunology Franchise with HLH Drug Approval

    Publicado: 27/12/2018
20. Learning to Thrive

    Publicado: 19/12/2018