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.
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.
Read ArticleCures 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)
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.
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.