Rare Disease Drug Repurposing
The findings of this qualitative study of RDNP repurposing suggest that several opportunities were associated with successful outcomes and can be considered to optimize systematic repurposing among RDNPs, external collaborators, and policymakers with the use of an interactive tool showcasing insights to facilitate data-driven drug repurposing.
Authors
- Sarah E. Bolden (ORCID: https://orcid.org/0000-0002-8992-1580)
- David C. Fajgenbaum (ORCID: https://orcid.org/0000-0002-7367-8184)
- Ania Korsunska (ORCID: https://orcid.org/0000-0003-3158-8436)
- Sally Nijim
- Joseph Zinski (ORCID: https://orcid.org/0000-0002-5120-5386)
- Mary Zuccato
- Mileva Repasky
Institutions
- Syracuse University (US)
- University of Pennsylvania (US)
Publication Details
- Journal
- JAMA Network Open
- Published
- 2025-05-05
- DOI
- https://doi.org/10.1001/jamanetworkopen.2025.8330
- Citations
- 5
- Primary Topic
- CRISPR and Genetic Engineering
- Type
- article
- Field-Weighted Citation Impact
- 4.03
Funders
- Fanconi Anemia Research Fund
- Barth Syndrome Foundation
- Foundation for Ichthyosis and Related Skin Types
- Myasthenia Gravis Foundation of America
- Hermansky-Pudlak Syndrome Network
- Muscular Dystrophy Association
- Nephcure Foundation
- CureSearch for Children's Cancer
- Child Neurology Foundation
- Cure HHT
- Chordoma Foundation
- RYR-1 Foundation
- Cholangiocarcinoma Foundation
- Beyond Batten Disease Foundation
- Campaign Urging Research for Eosinophilic Disease
- International Fibrodysplasia Ossificans Progressiva Association
- Glut1 Deficiency Foundation
- Foundation for Sarcoidosis Research
- Cure JM Foundation
- Cure CMD
- Academy of Finland