RARECast

548 Episodo

1. Leveraging Technology to Empower Patients and Decentralize Clinical Trials

   Publicado: 20/10/2022
2. Equipping The Next-Generation Rare Disease Patient Advocate

   Publicado: 13/10/2022
3. How a Family Raced from Diagnosis to Experimental Gene Therapy in Three Years

   Publicado: 6/10/2022
4. How One Advocate Went from Losing His Hearing to Being Heard

   Publicado: 29/9/2022
5. Building a Pipeline of Therapies to Treat Rare Mineralization Disorders

   Publicado: 22/9/2022
6. Advancing an Oral Alternative to Infused and Injected Therapies for HAE

   Publicado: 15/9/2022
7. Embracing the Promise of Patient-Centered Biotechnology

   Publicado: 8/9/2022
8. Learning to Live with Loss

   Publicado: 1/9/2022
9. Using Cryptocurrency to Tap into the Wisdom of Crowds

   Publicado: 25/8/2022
10. A Therapy for a Rare Neurodegenerative Disease Moves Towards Regulatory Review

    Publicado: 18/8/2022
11. Transforming the Treatment of Neuromuscular Diseases with Next-Gen Oligonucleotides

    Publicado: 11/8/2022
12. Teaching the Immune System to Let Medicines Do Their Job

    Publicado: 4/8/2022
13. Designing Clinical Trials with the Patient in Mind

    Publicado: 28/7/2022
14. Keeping Clinical Trials Running Smoothly

    Publicado: 21/7/2022
15. A Vision for Patient-Centric Gene Therapy Development

    Publicado: 14/7/2022
16. Using Genetic Testing to Address Disparities in Care for Kidney Disease

    Publicado: 7/7/2022
17. Why Reforms to the Accelerated Approval Pathway Threaten Rare Disease Drug Development

    Publicado: 30/6/2022
18. A Longstanding Academic-Nonprofit Collaboration Gives Rise to an ALS Drug Company

    Publicado: 16/6/2022
19. Targeting Regulatory RNA to Upregulate Gene Expression to Treat Rare Diseases

    Publicado: 9/6/2022
20. Developing a New Approach to Treat Rare, Autoimmune Conditions

    Publicado: 2/6/2022