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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.

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.

Study Finds Genetic Variant Among People Who Experience a Rare Recovery From ALS

July 30, 2024 - Grantee/Partner News

  • Rare Diseases Clinical Research Network (RDCRN)

Looking for the Common in Rare

July 20, 2024 - Grantee/Partner News

  • Our Impact on Rare Diseases

Using AI to Improve Detection of Rare Diseases

July 16, 2024 - Grantee/Partner News

  • Clinical and Translational Science Awards (CTSA) Program

Paving the Way for Ultrarare Bone Disease Treatment Trial

May 21, 2024 - NCATS News

  • Our Impact on Drug Discovery and Development
  • Therapeutics for Rare and Neglected Diseases (TRND)

Clinicians at the NIH Clinical Center will soon start testing an investigative drug for an ultrarare bone disease.

Last updated on February 19, 2025