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.
How Repurposing a Drug May Help Treat More Rare Gastrointestinal Diseases
June 29, 2026 - NCATS News
- Rare Diseases Clinical Research Network (RDCRN)
RDCRN researchers found that dupilumab — a drug used for asthma and chronic obstructive pulmonary disease — shows promise in treating eosinophilic gastritis.
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FDA Clears IND for Gene Therapy Candidate to Treat Rare Metabolic Disorder
May 21, 2026 - NCATS News
- Our Impact on Clinical Trials
- Our Impact on Drug Discovery and Development
- Our Impact on Rare Diseases
MMA-101, an NIH-supported AAV gene therapy for a rare metabolic disorder, has received FDA clearance to begin clinical testing.
Read ArticleResearchers 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.
2021 Rare Disease Day at NIH: Finding Hope Within Uncertainty
April 1, 2021 - NCATS News
The COVID-19 pandemic has profoundly affected nearly every aspect of the rare disease world. The greatest impacts of the pandemic, however, have been felt by the patients themselves and their families.