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.
Paving the Way for Ultrarare Bone Disease Treatment Trial
May 21, 2024 - NCATS News
- Our Impact on Drug Discovery and Development
- Therapeutics for Rare and Neglected Diseases (TRND)
Clinicians at the NIH Clinical Center will soon start testing an investigative drug for an ultrarare bone disease.
Read ArticleEveryLife Foundation Announces Scientific Workshop on ‘Ultra-Rare’ Diseases
May 16, 2024 - Media Coverage
- Our Impact on Rare Diseases
AI Is Revolutionizing Rare Disease Research, but a Lack of Diversity Undermines Its Full Potential
September 5, 2023 - Media Coverage
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.
Rare Disease Day Inspires, Offers Resources and Hope
April 14, 2023 - Grantee/Partner News
- Genetic and Rare Diseases (GARD) Information Center
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.
Benefiting From Orphan Drug and Rare Pediatric Disease Designations for Gene Therapy
March 27, 2023 - Media Coverage