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)
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.
Breaking Down Prednisone Too Quickly May Short-Circuit Its Benefit for Rare Neuromuscular Disorder
February 15, 2024 - NCATS News
- Rare Diseases Clinical Research Network (RDCRN)
Chemicals produced when the body breaks down the drug prednisone could indicate which patients with a rare muscle disorder were helped by the drug.
Newborn Screening for Rare Immune Disorders Led to Earlier Treatment, Improved Survival
November 20, 2023 - NCATS News
- Rare Diseases Clinical Research Network (RDCRN)
An NCATS-led Rare Diseases Clinical Research Network consortium changed what is known about severe combined immunodeficiency. Researchers developed lifesaving therapies for the disease and pioneered a screening test for newborns.
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. This additional screening could allow lifesaving treatments to be delivered to babies sooner.
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. Food and Drug Administration (FDA), it marked a key accomplishment for researchers in the NIH's Platform Vector Gene Therapy (PaVe-GT) project.