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.
WEST 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.
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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.
Read ArticlePushing for Better Clinical Trial Design for Rare Diseases
October 17, 2023 - Grantee/Partner News
New 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.
Gene Therapy: The Future Made Present for Addressing Rare Diseases
April 18, 2023 - Media Coverage
- Bespoke Gene Therapy Consortium (BGTC)
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.