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 Awards $15M+ to Baylor-Led Consortia Targeting Rare Bone and Metabolic Disorders
September 27, 2025 - Media Coverage
- Rare Diseases Clinical Research Network (RDCRN)
NIH Awards More Than $15 Million to Fund Two Rare Diseases Consortia
September 25, 2025 - Grantee/Partner News
- Rare Diseases Clinical Research Network (RDCRN)
Data Science Tools Will Speed Rare Disease Solutions
March 15, 2023 - NCATS News
- Genetic and Rare Diseases (GARD) Information Center
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.
Drug Repurposing Approaches Reveal HIV Drug Holds Promise Against a Rare, Disabling Muscle Disorder
November 30, 2021 - NCATS News
Using drug repurposing approaches, researchers from NCATS, Duke University and the University of Florida discovered that an existing antiviral drug used against HIV infection may be helpful in treating a form of dystonia.
NIH, FDA and 15 Private Organizations Join Forces to Increase Effective Gene Therapies for Rare Diseases
October 27, 2021 - NCATS News
- Bespoke Gene Therapy Consortium (BGTC)
- Platform Vector Gene Therapy (PaVe-GT)
NIH, FDA and 15 private organizations partnered to create the Bespoke Gene Therapy Consortium to speed development of FDA-approved gene therapies for rare diseases. The effort is co-led by NCATS and managed by FNIH.
NIH Study Suggests People with Rare Diseases Face Significantly Higher Health Care Costs
October 22, 2021 - NCATS News
Results from a collaborative pilot study suggest health care costs for each person with a rare disease are three to five times greater than the costs for those who do not have a rare disease.