RARECast

548 Avsnitt

1. Leveraging Technology to Empower Patients and Decentralize Clinical Trials

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

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

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

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

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

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

   Publicerades: 2022-09-08
8. Learning to Live with Loss

   Publicerades: 2022-09-01
9. Using Cryptocurrency to Tap into the Wisdom of Crowds

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

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

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

    Publicerades: 2022-08-04
13. Designing Clinical Trials with the Patient in Mind

    Publicerades: 2022-07-28
14. Keeping Clinical Trials Running Smoothly

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

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

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

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

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

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

    Publicerades: 2022-06-02