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)
Personalized CRISPR Therapy Successfully Treats Infant With Rare, Incurable CPS1 Deficiency
May 16, 2025 - Media Coverage
- Our Impact on Rare Diseases
- Somatic Cell Genome Editing (SCGE)
Infant With Rare, Incurable Disease Is First to Successfully Receive Personalized Gene Therapy Treatment
May 15, 2025 - Grantee/Partner News
- Our Impact on Rare Diseases
- Somatic Cell Genome Editing (SCGE)
Infant With Rare Incurable Disease Becomes First to Receive Successful Personalized Gene Therapy
May 15, 2025 - Media Coverage
- Our Impact on Rare Diseases
- Somatic Cell Genome Editing (SCGE)
Researchers Identify Pathway Responsible for Calciphylaxis, a Rare and Serious Condition
April 23, 2025 - Grantee/Partner News
- Clinical and Translational Science Awards (CTSA) Program
A Combination of Tests Shows Learning Capabilities of Nonverbal Children with Rare Brain Disorder
February 25, 2025 - NCATS News
- Our Impact on Rare Diseases
- Rare Diseases Clinical Research Network (RDCRN)
NCATS-funded scientists found that a pair of tests can measure nonverbal learning skills and show the cognitive abilities of children with Aicardi-Goutières syndrome. The tests could improve the support and therapy doctors provide.
AI Tool Helps Find Life-Saving Medicine for Rare Disease
February 5, 2025 - Grantee/Partner News
- Biomedical Data Translator (Translator)
- Our Impact on Rare Diseases