RARECast

512 Folgen

1. How a Drug Setback Became a Patient Community’s Gain

   Vom: 18.11.2022
2. Helping Regulators and Drug Developers Understand the Challenges of Living with Fabry Disease

   Vom: 10.11.2022
3. Addressing Racial Disparities in a Rare Blood Cancer

   Vom: 3.11.2022
4. How New Sequencing Technology Promises to Alter the Diagnostic Odyssey

   Vom: 27.10.2022
5. Leveraging Technology to Empower Patients and Decentralize Clinical Trials

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

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

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

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

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

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

    Vom: 8.9.2022
12. Learning to Live with Loss

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

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

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

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

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

    Vom: 28.7.2022
18. Keeping Clinical Trials Running Smoothly

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

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

    Vom: 7.7.2022