RARECast

512 Folgen

1. aTyr Pharma Seeks to Modulate Activity in FSHD and Other Rare Disease

   Vom: 28.6.2017
2. Summit Pursues A Different Approach to Treating Duchenne

   Vom: 21.6.2017
3. Delivering Enzyme Replacement Therapies Across the Blood-Brain Barrier

   Vom: 14.6.2017
4. Sangamo Advances Gene Editing Therapies for Multiple Rare Diseases into the Clinic

   Vom: 7.6.2017
5. Navigating the Challenges of Rare Disease Drug Development

   Vom: 31.5.2017
6. Accelerating Rare Disease Drug Discovery

   Vom: 24.5.2017
7. Experimental Rare Disease Therapy Wins New FDA Reg Med Designation

   Vom: 17.5.2017
8. How Rare Disease Patients Can Get their Voices Heard in Drug Discovery and Development

   Vom: 10.5.2017
9. One Patient's Difficult Road to a Rare Diagnosis

   Vom: 3.5.2017
10. How Researchers Diagnosed Four Patients with a Never-Before-Identified Rare Disease in a Day

    Vom: 26.4.2017
11. Stem Cell Gene Therapy Restores Immune System in Kids with SCID

    Vom: 19.4.2017
12. Rare Disease Advocates Push for Incentives to Drugmakers Pulled from Cures Act

    Vom: 12.4.2017
13. Non-Profit Drug Company Seeks to Advance Rare Disease Therapies

    Vom: 5.4.2017
14. Innovative Collaboration Model Drives Treatment for Rare Disease

    Vom: 29.3.2017
15. Girl with Rare Disease Inspires Search for Bone Marrow Donors

    Vom: 22.3.2017
16. A Quest for Tribe: Searching for Others with the Same Ultra-Rare Mutation

    Vom: 15.3.2017
17. Raising Awareness in a Town with a History of a Rare Disease

    Vom: 8.3.2017
18. Helping Rare Disease Patients Navigate the World of Information

    Vom: 1.3.2017
19. Overcoming the Challenges of Rare Disease Drug Development

    Vom: 22.2.2017
20. Moving Rare Disease Therapies from Hope to Reality

    Vom: 15.2.2017