RARECast

548 Odcinki

1. Leveraging Technology to Empower Patients and Decentralize Clinical Trials

   Opublikowany: 20.10.2022
2. Equipping The Next-Generation Rare Disease Patient Advocate

   Opublikowany: 13.10.2022
3. How a Family Raced from Diagnosis to Experimental Gene Therapy in Three Years

   Opublikowany: 6.10.2022
4. How One Advocate Went from Losing His Hearing to Being Heard

   Opublikowany: 29.09.2022
5. Building a Pipeline of Therapies to Treat Rare Mineralization Disorders

   Opublikowany: 22.09.2022
6. Advancing an Oral Alternative to Infused and Injected Therapies for HAE

   Opublikowany: 15.09.2022
7. Embracing the Promise of Patient-Centered Biotechnology

   Opublikowany: 8.09.2022
8. Learning to Live with Loss

   Opublikowany: 1.09.2022
9. Using Cryptocurrency to Tap into the Wisdom of Crowds

   Opublikowany: 25.08.2022
10. A Therapy for a Rare Neurodegenerative Disease Moves Towards Regulatory Review

    Opublikowany: 18.08.2022
11. Transforming the Treatment of Neuromuscular Diseases with Next-Gen Oligonucleotides

    Opublikowany: 11.08.2022
12. Teaching the Immune System to Let Medicines Do Their Job

    Opublikowany: 4.08.2022
13. Designing Clinical Trials with the Patient in Mind

    Opublikowany: 28.07.2022
14. Keeping Clinical Trials Running Smoothly

    Opublikowany: 21.07.2022
15. A Vision for Patient-Centric Gene Therapy Development

    Opublikowany: 14.07.2022
16. Using Genetic Testing to Address Disparities in Care for Kidney Disease

    Opublikowany: 7.07.2022
17. Why Reforms to the Accelerated Approval Pathway Threaten Rare Disease Drug Development

    Opublikowany: 30.06.2022
18. A Longstanding Academic-Nonprofit Collaboration Gives Rise to an ALS Drug Company

    Opublikowany: 16.06.2022
19. Targeting Regulatory RNA to Upregulate Gene Expression to Treat Rare Diseases

    Opublikowany: 9.06.2022
20. Developing a New Approach to Treat Rare, Autoimmune Conditions

    Opublikowany: 2.06.2022