Skip to main content

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.

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.

Using AI to Improve Detection of Rare Diseases

July 16, 2024 - Grantee/Partner News

  • Clinical and Translational Science Awards (CTSA) Program

At a Symposium on Rare Diseases, Smiles Were in Abundance

March 28, 2024 - Grantee/Partner News

  • Our Impact on Rare Diseases

Pushing for Better Clinical Trial Design for Rare Diseases

October 17, 2023 - Grantee/Partner News

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, 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.

Last updated on September 8, 2025