Skip to main content
HHS Logo U.S. Department of Health & Human Services NIH Logo National Institutes of Health

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.

Cartoon of a double-helix DNA polymer and cells.

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
Rare Diseases: Individually Rare, Collectively Common. NCATS is developing new approaches to diagnose and treat people with rare diseases more quickly. NCATS logo. Resources. Providing high-quality, widely used resources like the Genetic and Rare Diseases Information Center (GARD), to educate, engage and empower the rare diseases community. Getting an accurate rare disease diagnosis can take more than 6 years, on average, leading to higher healthcare costs. GARD staff have answered about 120,000 questions.

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 Article

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.

Read Article

Global Patient Registries Can Advance Opportunities for Rare Disease Research

December 14, 2023 - Media Coverage

  • Rare Diseases Registry Program (RaDaR)
Read Article

Rare Syndrome After COVID Vaccines Described

December 3, 2023 - Media Coverage

  • Clinical and Translational Science Awards (CTSA) Program
Read Article

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.

Read Article

Pushing for Better Clinical Trial Design for Rare Diseases

October 17, 2023 - Grantee/Partner News

Read Article

AI Is Revolutionizing Rare Disease Research, but a Lack of Diversity Undermines Its Full Potential

September 5, 2023 - Media Coverage

Read Article

Last updated on June 13, 2024