RARECast

548 Episodio

1. Leveraging Technology to Empower Patients and Decentralize Clinical Trials

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

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

   Pubblicato: 06/10/2022
4. How One Advocate Went from Losing His Hearing to Being Heard

   Pubblicato: 29/09/2022
5. Building a Pipeline of Therapies to Treat Rare Mineralization Disorders

   Pubblicato: 22/09/2022
6. Advancing an Oral Alternative to Infused and Injected Therapies for HAE

   Pubblicato: 15/09/2022
7. Embracing the Promise of Patient-Centered Biotechnology

   Pubblicato: 08/09/2022
8. Learning to Live with Loss

   Pubblicato: 01/09/2022
9. Using Cryptocurrency to Tap into the Wisdom of Crowds

   Pubblicato: 25/08/2022
10. A Therapy for a Rare Neurodegenerative Disease Moves Towards Regulatory Review

    Pubblicato: 18/08/2022
11. Transforming the Treatment of Neuromuscular Diseases with Next-Gen Oligonucleotides

    Pubblicato: 11/08/2022
12. Teaching the Immune System to Let Medicines Do Their Job

    Pubblicato: 04/08/2022
13. Designing Clinical Trials with the Patient in Mind

    Pubblicato: 28/07/2022
14. Keeping Clinical Trials Running Smoothly

    Pubblicato: 21/07/2022
15. A Vision for Patient-Centric Gene Therapy Development

    Pubblicato: 14/07/2022
16. Using Genetic Testing to Address Disparities in Care for Kidney Disease

    Pubblicato: 07/07/2022
17. Why Reforms to the Accelerated Approval Pathway Threaten Rare Disease Drug Development

    Pubblicato: 30/06/2022
18. A Longstanding Academic-Nonprofit Collaboration Gives Rise to an ALS Drug Company

    Pubblicato: 16/06/2022
19. Targeting Regulatory RNA to Upregulate Gene Expression to Treat Rare Diseases

    Pubblicato: 09/06/2022
20. Developing a New Approach to Treat Rare, Autoimmune Conditions

    Pubblicato: 02/06/2022