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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 Research Shows Female OTC Carriers (Heterozygous Females) Face More Symptoms, Risks Than Expected

June 28, 2024 - Grantee/Partner News

  • Rare Diseases Clinical Research Network (RDCRN)

Children’s Hospital of Philadelphia Researchers Find Some Patients With Aicardi Goutières Syndrome Have Normal IQ

June 24, 2024 - Grantee/Partner News

  • Rare Diseases Clinical Research Network (RDCRN)

NCATS-Funded Rare Disease Researchers Provide Key Data, Help Build New Kidney Atlas

February 29, 2024 - NCATS News

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

NEPTUNE’s research has helped us learn more about the biology of both rare and common kidney diseases. It has also led to better ways to diagnose and treat these conditions.

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.

Propelling Cures With Genetics Research: Interview With Joni L. Rutter, PhD, Director of the National Center for Advancing Translational Sciences at NIH

December 14, 2023 - Grantee/Partner News

  • 3-D Tissue Bioprinting
  • Bespoke Gene Therapy Consortium (BGTC)
  • Clinical and Translational Science Awards (CTSA) Program
  • New Therapeutic Uses (NTU)
  • Platform Vector Gene Therapy (PaVe-GT)
  • Rare Diseases Clinical Research Network (RDCRN)
  • Somatic Cell Genome Editing (SCGE)
  • Therapeutics for Rare and Neglected Diseases (TRND)
  • Tissue Chip for Drug Screening

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.

Last updated on June 13, 2024