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 ArticleNew 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.
Genetics Experts Support Adding Hundreds of Treatable Rare Diseases to Newborn Screening
May 9, 2023 - NCATS News
- Clinical and Translational Science Awards (CTSA) Program
A study found that 88% of experts surveyed on rare diseases agreed that DNA testing to reveal treatable genetic disorders should be available for all infants. This additional screening could allow lifesaving treatments to be delivered to babies sooner.
NIH Gene Therapy Team Reveals Its Path to FDA Orphan Drug and Rare Pediatric Disease Designations
March 29, 2023 - NCATS News
- Our Impact on Rare Diseases
- Our Impact on Research Operations
- Platform Vector Gene Therapy (PaVe-GT)
When NCATS received an Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA), it marked a key accomplishment for researchers in the NIH's Platform Vector Gene Therapy (PaVe-GT) project.
Rare Disease Day at NIH 2023: Putting Hope into Action
March 21, 2023 - NCATS News
Energy, excitement and hope guided Rare Disease Day at NIH 2023, which included inspiring stories from people with rare diseases, research updates, exhibits and poster sessions, and networking.
Data Science Tools Will Speed Rare Disease Solutions
March 15, 2023 - NCATS News
- Genetic and Rare Diseases (GARD) Information Center
NLM musings from the Mezzanine. Data science tools will speed rare disease solutions.
Rare Disease Day at NIH 2022: Shining a Light on Patient Perspectives
May 11, 2022 - NCATS News
- Genetic and Rare Diseases (GARD) Information Center
For the second year, the annual Rare Disease Day at NIH 2022 event was held virtually due to the pandemic, but that didn’t stop patients and researchers from coming together to share their stories and highlight research efforts.