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 ArticleNavigating a Rare Disease
June 3, 2025 - Grantee/Partner News
- Genetic and Rare Diseases (GARD) Information Center
- Rare Diseases Clinical Research Network (RDCRN)
Infant with Rare, Incurable Disease is First to Successfully Receive Personalized Gene Therapy Treatment
May 28, 2025 - NCATS News
- Our Impact on Rare Diseases
- Somatic Cell Genome Editing (SCGE)
NIH-supported gene-editing platform lays groundwork to rapidly develop treatments for other rare genetic diseases.
Repairing a Broken Code of Life: In CRISPR Breakthrough, Rewriting the Misspelling of Infant’s DNA Cures Him of Rare Liver Disease
May 16, 2025 - Grantee/Partner News
- Our Impact on Rare Diseases
- Somatic Cell Genome Editing (SCGE)
Personalized CRISPR Therapy Successfully Treats Infant With Rare, Incurable CPS1 Deficiency
May 16, 2025 - Media Coverage
- Our Impact on Rare Diseases
- Somatic Cell Genome Editing (SCGE)
Infant With Rare, Incurable Disease Is First to Successfully Receive Personalized Gene Therapy Treatment
May 15, 2025 - Grantee/Partner News
- Our Impact on Rare Diseases
- Somatic Cell Genome Editing (SCGE)
Infant With Rare Incurable Disease Becomes First to Receive Successful Personalized Gene Therapy
May 15, 2025 - Media Coverage
- Our Impact on Rare Diseases
- Somatic Cell Genome Editing (SCGE)