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)
Researchers Create 3-D Model for Rare Neuromuscular Disorders, Setting Stage for Clinical Trial
April 19, 2022 - NCATS News
- Small Business (SBIR)
- Tissue Chip for Drug Screening
Partially funded by a Small Business Innovation Research grant from NCATS, Hesperos, Inc., researchers created a tissue chip model that mimics the biology of two rare neuromuscular disorders, setting the stage for testing in a clinical trial.
Drug Repurposing Approaches Reveal HIV Drug Holds Promise Against a Rare, Disabling Muscle Disorder
November 30, 2021 - NCATS News
Using drug repurposing approaches, researchers from NCATS, Duke University and the University of Florida discovered that an existing antiviral drug used against HIV infection may be helpful in treating a form of dystonia.
NIH, FDA and 15 Private Organizations Join Forces to Increase Effective Gene Therapies for Rare Diseases
October 27, 2021 - NCATS News
- Bespoke Gene Therapy Consortium (BGTC)
- Platform Vector Gene Therapy (PaVe-GT)
NIH, FDA and 15 private organizations partnered to create the Bespoke Gene Therapy Consortium to speed development of FDA-approved gene therapies for rare diseases. The effort is co-led by NCATS and managed by FNIH.
NIH Study Suggests People with Rare Diseases Face Significantly Higher Health Care Costs
October 22, 2021 - NCATS News
Results from a collaborative pilot study suggest health care costs for each person with a rare disease are three to five times greater than the costs for those who do not have a rare disease.
With a Nudge from AI, Ketamine Emerges as a Potential Rare Disease Treatment
August 5, 2021 - Media Coverage
- Biomedical Data Translator (Translator)
Genetic Analysis Suggests Dilated Cardiomyopathy Therapies May Work for Rare Peripartum Cardiomyopathy
May 11, 2021 - NCATS News
- Clinical and Translational Science Awards (CTSA) Program
Analyzing genetic information drawn from patients and multiple databases, CTSA Program researchers discovered similarities between nonischemic dilated cardiomyopathy (DCM) and peripartum cardiomyopathy (PPCM).