RARECast

548 Episoder

1. Choosing the Right Viral Vector for a Gene Therapy

   Publisert: 1.4.2021
2. A Software Platform to Give Patients the Tools to Build Treatments for Rare Genetic Diseases

   Publisert: 25.3.2021
3. Side Effects: The Toll a Rare Disease Can Take on a Family in Pursuit of a Cure

   Publisert: 19.3.2021
4. Realizing the Potential of CRISPR Gene Editing

   Publisert: 12.3.2021
5. Understanding the Economic Toll of Rare Disease in the United States

   Publisert: 4.3.2021
6. The Promise of Gene-Based Therapies for Neurodegenerative Conditions

   Publisert: 25.2.2021
7. Moving Beyond Viral Vectors for Gene Therapies

   Publisert: 19.2.2021
8. Acquisitions Help Jazz Build Toward Commercial Crescendo

   Publisert: 12.2.2021
9. Powerful Gene Editing Approach Offers the Promise of Correcting a Range of Rare Diseases

   Publisert: 5.2.2021
10. A Child's-Eye-View of Clinical Trials

    Publisert: 29.1.2021
11. Using Nanoliposomes to Make Cancer Therapies Safer and More Effective

    Publisert: 21.1.2021
12. Accelerating Treatments for Rare Disease through Data Sharing

    Publisert: 14.1.2021
13. Treating the Root Cause of Sickle Cell Disease

    Publisert: 8.1.2021
14. Targeting a Common Pathway in Genetic Forms of Obesity

    Publisert: 30.12.2020
15. After Pruning Orchard, Gaspar Focuses on High Value Opportunities

    Publisert: 23.12.2020
16. Writing a New Chapter of Genetic Medicine

    Publisert: 17.12.2020
17. How an Ultra-Rare Disease Patient Organization Drove Research to a Treatment

    Publisert: 10.12.2020
18. With a New Identity, Travere Thearpeutics Seeks to Find Its True Path

    Publisert: 4.12.2020
19. Making Gene Therapies Accessible to Patient with Ultra-Rare Conditions

    Publisert: 26.11.2020
20. Using RNA Therapies to Target Inherited Retinal Diseases

    Publisert: 20.11.2020