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.
NCATS Enables a Drug’s Path to FDA Approval for Duchenne Muscular Dystrophy
March 15, 2024 - NCATS News
- Our Impact on Drug Discovery and Development
- Our Impact on Rare Diseases
- Therapeutics for Rare and Neglected Diseases (TRND)
NCATS scientists conducted laboratory studies and provided guidance in the development of a drug for the rare muscle disease Duchenne muscular dystrophy.
Propelling Cures With Genetics Research: Interview With Joni L. Rutter, PhD, Director of the National Center for Advancing Translational Sciences at NIH
December 14, 2023 - Grantee/Partner News
- 3-D Tissue Bioprinting
- Bespoke Gene Therapy Consortium (BGTC)
- Clinical and Translational Science Awards (CTSA) Program
- New Therapeutic Uses (NTU)
- Platform Vector Gene Therapy (PaVe-GT)
- Rare Diseases Clinical Research Network (RDCRN)
- Somatic Cell Genome Editing (SCGE)
- Therapeutics for Rare and Neglected Diseases (TRND)
- Tissue Chip for Drug Screening
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.
Blood Pressure Drug Could Treat Lebers
April 4, 2023 - Media Coverage
- Therapeutics for Rare and Neglected Diseases (TRND)
FDA-Approved Drug Shows Promise in Laboratory Models for Leber Congenital Amaurosis Type 10
April 2, 2023 - Media Coverage
- Therapeutics for Rare and Neglected Diseases (TRND)
Old Drugs, New Uses: Advances in Drug Repurposing and the Federal Outlook
March 14, 2023 - Media Coverage
- Bridging Interventional Development Gaps (BrIDGs)
- New Therapeutic Uses (NTU)
- Therapeutics for Rare and Neglected Diseases (TRND)
Sugar Molecule Could Resolve Rare Diseases
February 28, 2023 - Grantee/Partner News
- Therapeutics for Rare and Neglected Diseases (TRND)
JAMA Neurology Publishes Positive Pivotal Clinical Trial with Vamorolone in Duchenne Muscular Dystrophy
September 1, 2022 - Media Coverage
- Therapeutics for Rare and Neglected Diseases (TRND)