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.
Read Article
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 ArticleWEST AI Algorithm May Help Speed Diagnosis of Rare Diseases
April 14, 2026 - NCATS News
- Clinical and Translational Science Awards (CTSA) Program
- Our Impact on Rare Diseases
Rare diseases can take years to diagnose. WEST — a new AI algorithm — aims to cut that diagnostic odyssey by learning from complete and incomplete real-world EHR data.
Rare Disease Day at NIH 2026: Paving the Way to a Brighter Future for All Americans
March 31, 2026 - NCATS News
- Our Impact on Rare Diseases
The rare diseases community came together at the NIH Natcher Conference Center and virtually to celebrate Rare Disease Day at NIH, highlighting inspiring stories, impactful research advances, and new possibilities for the future.
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.
Cures 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)
Children’s Hospital of Philadelphia Marks One-Year Anniversary of World’s First Personalized CRISPR Gene Therapy for Child with Rare Genetic Disease
February 24, 2026 - Grantee/Partner News
- Somatic Cell Genome Editing (SCGE)
New Initiative Aims to Revive Stalled Rare Disease Therapies
February 24, 2026 - Media Coverage
- Our Impact on Rare Diseases