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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.

Infant With Rare Incurable Disease Becomes First to Receive Successful Personalized Gene Therapy

May 15, 2025 - Media Coverage

  • Our Impact on Rare Diseases
  • Somatic Cell Genome Editing (SCGE)

Researchers Identify Pathway Responsible for Calciphylaxis, a Rare and Serious Condition

April 23, 2025 - Grantee/Partner News

  • Clinical and Translational Science Awards (CTSA) Program

A Combination of Tests Shows Learning Capabilities of Nonverbal Children with Rare Brain Disorder

February 25, 2025 - NCATS News

  • Our Impact on Rare Diseases
  • Rare Diseases Clinical Research Network (RDCRN)

NCATS-funded scientists found that a pair of tests can measure nonverbal learning skills and show the cognitive abilities of children with Aicardi-Goutières syndrome. The tests could improve the support and therapy doctors provide.

AI Tool Helps Find Life-Saving Medicine for Rare Disease

February 5, 2025 - Grantee/Partner News

  • Biomedical Data Translator (Translator)
  • Our Impact on Rare Diseases

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.

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.

Last updated on September 8, 2025