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.

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.
Read ArticleStudy Finds Genetic Variant Among People Who Experience a Rare Recovery From ALS
July 30, 2024 - Grantee/Partner News
- Rare Diseases Clinical Research Network (RDCRN)
Bridging the Gap: Why Translational Research Is Key to Unlocking Cell & Gene Therapy Innovation
July 22, 2024 - Grantee/Partner News
- Rare Diseases Clinical Research Network (RDCRN)
New Research Shows Female OTC Carriers (Heterozygous Females) Face More Symptoms, Risks Than Expected
June 28, 2024 - Grantee/Partner News
- Rare Diseases Clinical Research Network (RDCRN)
Children’s Hospital of Philadelphia Researchers Find Some Patients With Aicardi Goutières Syndrome Have Normal IQ
June 24, 2024 - Grantee/Partner News
- Rare Diseases Clinical Research Network (RDCRN)
NCATS-Funded Rare Disease Researchers Provide Key Data, Help Build New Kidney Atlas
February 29, 2024 - NCATS News
- Our Impact on Rare Diseases
- Rare Diseases Clinical Research Network (RDCRN)
NEPTUNE’s research has helped us learn more about the biology of both rare and common kidney diseases. It has also led to better ways to diagnose and treat these conditions.
Breaking Down Prednisone Too Quickly May Short-Circuit Its Benefit for Rare Neuromuscular Disorder
February 15, 2024 - NCATS News
- Rare Diseases Clinical Research Network (RDCRN)
Chemicals produced when the body breaks down the drug prednisone could indicate which patients with a rare muscle disorder were helped by the drug.
Propelling Cures With Genetics Research: Interview With Joni L. Rutter, PhD, Director of the National Center for Advancing Translational Sciences at NIH
December 14, 2023 - Grantee/Partner News
- 3-D Tissue Bioprinting
- Bespoke Gene Therapy Consortium (BGTC)
- Clinical and Translational Science Awards (CTSA) Program
- New Therapeutic Uses (NTU)
- Platform Vector Gene Therapy (PaVe-GT)
- Rare Diseases Clinical Research Network (RDCRN)
- Somatic Cell Genome Editing (SCGE)
- Therapeutics for Rare and Neglected Diseases (TRND)
- Tissue Chip for Drug Screening