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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.

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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.

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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.

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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)
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Looking for the Common in Rare

July 20, 2024 - Grantee/Partner News

  • Our Impact on Rare Diseases
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Using AI to Improve Detection of Rare Diseases

July 16, 2024 - Grantee/Partner News

  • Clinical and Translational Science Awards (CTSA) Program
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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.

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EveryLife Foundation Announces Scientific Workshop on ‘Ultra-Rare’ Diseases

May 16, 2024 - Media Coverage

  • Our Impact on Rare Diseases
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Every Cure Gets $48 Million for AI-Powered Rare Disease Research

May 14, 2024 - Grantee/Partner News

  • Clinical and Translational Science Awards (CTSA) Program
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Last updated on June 13, 2024