RARECast

548 Folgen

1. Leveraging Technology to Empower Patients and Decentralize Clinical Trials

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

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

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

   Vom: 29.9.2022
5. Building a Pipeline of Therapies to Treat Rare Mineralization Disorders

   Vom: 22.9.2022
6. Advancing an Oral Alternative to Infused and Injected Therapies for HAE

   Vom: 15.9.2022
7. Embracing the Promise of Patient-Centered Biotechnology

   Vom: 8.9.2022
8. Learning to Live with Loss

   Vom: 1.9.2022
9. Using Cryptocurrency to Tap into the Wisdom of Crowds

   Vom: 25.8.2022
10. A Therapy for a Rare Neurodegenerative Disease Moves Towards Regulatory Review

    Vom: 18.8.2022
11. Transforming the Treatment of Neuromuscular Diseases with Next-Gen Oligonucleotides

    Vom: 11.8.2022
12. Teaching the Immune System to Let Medicines Do Their Job

    Vom: 4.8.2022
13. Designing Clinical Trials with the Patient in Mind

    Vom: 28.7.2022
14. Keeping Clinical Trials Running Smoothly

    Vom: 21.7.2022
15. A Vision for Patient-Centric Gene Therapy Development

    Vom: 14.7.2022
16. Using Genetic Testing to Address Disparities in Care for Kidney Disease

    Vom: 7.7.2022
17. Why Reforms to the Accelerated Approval Pathway Threaten Rare Disease Drug Development

    Vom: 30.6.2022
18. A Longstanding Academic-Nonprofit Collaboration Gives Rise to an ALS Drug Company

    Vom: 16.6.2022
19. Targeting Regulatory RNA to Upregulate Gene Expression to Treat Rare Diseases

    Vom: 9.6.2022
20. Developing a New Approach to Treat Rare, Autoimmune Conditions

    Vom: 2.6.2022