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.
NIH, AlphaRose Study Advances Personalised Therapy Approach for Rare Genetic Disorder
July 16, 2026 - Media Coverage
- Our Impact on Drug Discovery and Development
- Our Impact on Rare Diseases
FDA OKs First-in-Human Trial for BGTC-Supported Rare Disease Gene Therapy
July 7, 2026 - Media Coverage
- Bespoke Gene Therapy Consortium (BGTC)
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.
Researchers Create 3-D Model for Rare Neuromuscular Disorders, Setting Stage for Clinical Trial
April 19, 2022 - NCATS News
- Small Business (SBIR)
- Tissue Chip for Drug Screening
Partially funded by a Small Business Innovation Research grant from NCATS, Hesperos, Inc., researchers created a tissue chip model that mimics the biology of two rare neuromuscular disorders, setting the stage for testing in a clinical trial.
2021 Rare Disease Day at NIH: Finding Hope Within Uncertainty
April 1, 2021 - NCATS News
The COVID-19 pandemic has profoundly affected nearly every aspect of the rare disease world. The greatest impacts of the pandemic, however, have been felt by the patients themselves and their families.