RARECast

548 Episodo

1. Choosing the Right Viral Vector for a Gene Therapy

   Publicado: 1/4/2021
2. A Software Platform to Give Patients the Tools to Build Treatments for Rare Genetic Diseases

   Publicado: 25/3/2021
3. Side Effects: The Toll a Rare Disease Can Take on a Family in Pursuit of a Cure

   Publicado: 19/3/2021
4. Realizing the Potential of CRISPR Gene Editing

   Publicado: 12/3/2021
5. Understanding the Economic Toll of Rare Disease in the United States

   Publicado: 4/3/2021
6. The Promise of Gene-Based Therapies for Neurodegenerative Conditions

   Publicado: 25/2/2021
7. Moving Beyond Viral Vectors for Gene Therapies

   Publicado: 19/2/2021
8. Acquisitions Help Jazz Build Toward Commercial Crescendo

   Publicado: 12/2/2021
9. Powerful Gene Editing Approach Offers the Promise of Correcting a Range of Rare Diseases

   Publicado: 5/2/2021
10. A Child's-Eye-View of Clinical Trials

    Publicado: 29/1/2021
11. Using Nanoliposomes to Make Cancer Therapies Safer and More Effective

    Publicado: 21/1/2021
12. Accelerating Treatments for Rare Disease through Data Sharing

    Publicado: 14/1/2021
13. Treating the Root Cause of Sickle Cell Disease

    Publicado: 8/1/2021
14. Targeting a Common Pathway in Genetic Forms of Obesity

    Publicado: 30/12/2020
15. After Pruning Orchard, Gaspar Focuses on High Value Opportunities

    Publicado: 23/12/2020
16. Writing a New Chapter of Genetic Medicine

    Publicado: 17/12/2020
17. How an Ultra-Rare Disease Patient Organization Drove Research to a Treatment

    Publicado: 10/12/2020
18. With a New Identity, Travere Thearpeutics Seeks to Find Its True Path

    Publicado: 4/12/2020
19. Making Gene Therapies Accessible to Patient with Ultra-Rare Conditions

    Publicado: 26/11/2020
20. Using RNA Therapies to Target Inherited Retinal Diseases

    Publicado: 20/11/2020