RARECast

512 Folgen

1. Why Reforms to the Accelerated Approval Pathway Threaten Rare Disease Drug Development

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

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

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

   Vom: 2.6.2022
5. Bridging the Gap Between Basic and Commercial Research for Rare Disease

   Vom: 26.5.2022
6. Stoking Functional Copies of Genes to Compensate for Mutated Ones

   Vom: 19.5.2022
7. Targeting ALS with Synergistic Combinations of Therapies

   Vom: 13.5.2022
8. How a Polish Rare Disease Organizations Is Helping Its Ukrainian Neighbors

   Vom: 5.5.2022
9. Powering a New Era of Genetic Medicine

   Vom: 28.4.2022
10. Searching for a Diagnosis

    Vom: 21.4.2022
11. A Novel Way to Deliver Rare Disease Therapies

    Vom: 14.4.2022
12. Bringing Diversity, Equity, and Inclusion to the Books We Read

    Vom: 7.4.2022
13. From Mila to Millions: Scaling N of 1 Therapies

    Vom: 31.3.2022
14. Genetically Modifying Patients’ Skin Cells to Treat Rare Diseases

    Vom: 24.3.2022
15. Targeting Rare, Immunologic Disorders

    Vom: 18.3.2022
16. Harnessing a Natural Mechanism to Silence Disease

    Vom: 10.3.2022
17. Advancing Precision Medicine with Patient Data

    Vom: 3.3.2022
18. Pregnancy and Motherhood for a Woman with SMA

    Vom: 24.2.2022
19. Steps to Improve Data Gathering of Rare Diseases

    Vom: 17.2.2022
20. Rare Patients Learn to Make Their Voices Heard

    Vom: 10.2.2022