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.
New Path for a Gene Therapy Trial at NIH for a Rare Metabolic Disease
November 19, 2024 - NCATS News
- Bespoke Gene Therapy Consortium (BGTC)
- Our Impact on Clinical Trials
- Our Impact on Drug Discovery and Development
- Our Impact on Rare Diseases
- Platform Vector Gene Therapy (PaVe-GT)
Scientists at NCATS and other NIH institutes and centers are teaming up to advance a gene therapy treatment for MMA, a rare and potentially life-threatening genetic disorder.
Read Article
Easing the Burden of Rare Diseases Through Translational Science
November 12, 2024 - NCATS News
- Our Impact on Rare Diseases
NCATS Director Joni Rutter discusses how NCATS is addressing scientific and operational roadblocks that slow progress for rare diseases research and treatments.
Read ArticleRare Disease Day at NIH 2024: Showcasing Voices and New Opportunities
April 23, 2024 - NCATS News
- Our Impact on Rare Diseases
The rare diseases community came together at Rare Disease Day at NIH to share inspiring stories, impactful research advances and exciting new possibilities for the future.
Families, Researchers Share Insights at Rare Disease Day
April 12, 2024 - Grantee/Partner News
- Our Impact on Rare Diseases
At a Symposium on Rare Diseases, Smiles Were in Abundance
March 28, 2024 - Grantee/Partner News
- Our Impact on Rare Diseases
Sharing Science and Stories at Rare Disease Day Symposium
March 7, 2024 - Grantee/Partner News
- Our Impact on Rare Diseases
NCATS-Funded Rare Disease Researchers Provide Key Data, Help Build New Kidney Atlas
February 29, 2024 - NCATS News
- Our Impact on Rare Diseases
- Rare Diseases Clinical Research Network (RDCRN)
NEPTUNE’s research has helped us learn more about the biology of both rare and common kidney diseases. It has also led to better ways to diagnose and treat these conditions.
Communicating About Rare Diseases
February 28, 2024 - NCATS News
- Our Impact on Rare Diseases
NCATS Director Joni Rutter penned a guest post for NIH’s Science, Health, and Public Trust blog on why communicating about rare diseases research is important and what to consider when sharing info