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.
Transparency and Early Planning Are Key in Increasing Efficiency of Gene Therapy Development for Rare Diseases
February 26, 2026 - NCATS News
- Platform Vector Gene Therapy (PaVe-GT)
PaVe-GT scientists underscore strategy, framework and early interaction with the FDA to align preclinical and clinical efforts in AAV gene therapy translation.
Read ArticleCures Within Reach Selected Three Clinical Trials in Rare/Ultra-Rare Diseases to Validate AI Predictions
February 25, 2026 - Grantee/Partner News
- Biomedical Data Translator (Translator)
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.
Global Patient Registries Can Advance Opportunities for Rare Disease Research
December 14, 2023 - Media Coverage
- Rare Diseases Registry Program (RaDaR)
Rare Syndrome After COVID Vaccines Described
December 1, 2023 - Media Coverage
- Clinical and Translational Science Awards (CTSA) Program
Newborn Screening for Rare Immune Disorders Led to Earlier Treatment, Improved Survival
November 20, 2023 - NCATS News
- Rare Diseases Clinical Research Network (RDCRN)
An NCATS-led Rare Diseases Clinical Research Network consortium changed what is known about severe combined immunodeficiency. Researchers developed lifesaving therapies for the disease and pioneered a screening test for newborns.
Pushing for Better Clinical Trial Design for Rare Diseases
October 17, 2023 - Grantee/Partner News
New 3-D Models Offer Insights Into the Biology of a Rare, Devastating Neurological Disease
July 28, 2023 - NCATS News
- 3-D Tissue Bioprinting
- Therapeutics for Rare and Neglected Diseases (TRND)
NCATS scientists created human cell–based models of a rare disorder, NGLY1 deficiency, to learn more about the disease’s development and biology.