News
Explore the latest stories and publications from NCATS and our partners, see upcoming events, or explore the variety of resources we provide to better understand translational science and our work.
News
Read the latest news from NCATS and its collaborators whose research is supported through the Center’s programs, or follow coverage of NCATS' translational research activities through national and local media.
NIH, AlphaRose Study Advances Personalised Therapy Approach for Rare Genetic Disorder
July 16, 2026 - Media Coverage
- Our Impact on Drug Discovery and Development
- Our Impact on Rare Diseases
FDA OKs First-in-Human Trial for BGTC-Supported Rare Disease Gene Therapy
July 7, 2026 - Media Coverage
- Bespoke Gene Therapy Consortium (BGTC)
Genome Editing Corrected Rare Brain Mutations in Mice. Could It Help Fight Neurological Diseases?
July 21, 2025 - Grantee/Partner News
- Somatic Cell Genome Editing (SCGE)
Experimental Compound Offers Potential Treatment for Rare, Often Fatal, Childhood Disease
July 9, 2025 - Grantee/Partner News
- Clinical and Translational Science Awards (CTSA) Program
Research Moves Slowly. Rare Diseases Don’t — So Patients Aren’t Waiting
June 28, 2025 - Media Coverage
- Our Impact on Rare Diseases
Navigating a Rare Disease
June 3, 2025 - Grantee/Partner News
- Genetic and Rare Diseases (GARD) Information Center
- Rare Diseases Clinical Research Network (RDCRN)
Infant with Rare, Incurable Disease is First to Successfully Receive Personalized Gene Therapy Treatment
May 28, 2025 - NCATS News
- Our Impact on Rare Diseases
- Somatic Cell Genome Editing (SCGE)
NIH-supported gene-editing platform lays groundwork to rapidly develop treatments for other rare genetic diseases.
Repairing a Broken Code of Life: In CRISPR Breakthrough, Rewriting the Misspelling of Infant’s DNA Cures Him of Rare Liver Disease
May 16, 2025 - Grantee/Partner News
- Our Impact on Rare Diseases
- Somatic Cell Genome Editing (SCGE)