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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.

With a Nudge from AI, Ketamine Emerges as a Potential Rare Disease Treatment

August 5, 2021 - Media Coverage

  • Biomedical Data Translator (Translator)

Genetic Analysis Suggests Dilated Cardiomyopathy Therapies May Work for Rare Peripartum Cardiomyopathy

May 11, 2021 - NCATS News

  • Clinical and Translational Science Awards (CTSA) Program

Analyzing genetic information drawn from patients and multiple databases, CTSA Program researchers discovered similarities between nonischemic dilated cardiomyopathy (DCM) and peripartum cardiomyopathy (PPCM).

2021 Rare Disease Day at NIH: Finding Hope Within Uncertainty

April 1, 2021 - NCATS News

The COVID-19 pandemic has profoundly affected nearly every aspect of the rare disease world. The greatest impacts of the pandemic, however, have been felt by the patients themselves and their families.

NCATS Researchers’ New Approach to Measure Gene Activity Could Speed Search for Rare Disease Therapies

March 23, 2021 - NCATS News

  • Our Impact on Research Operations

NCATS scientists design an innovative test to find potential treatments for Charcot-Marie-Tooth disease (CMT), one of the most common inherited neurological diseases, opening new pathways for rare diseases research.

PaVe-GT: Collaborative NIH Effort Aimed at Creating a Gene Therapy Playbook, Making Rare Disease Treatments More Accessible

November 30, 2020 - NCATS News

  • Our Impact on Rare Diseases
  • Platform Vector Gene Therapy (PaVe-GT)

The Platform Vector Gene Therapy (PaVe-GT) initiative aims to make gene therapy development and clinical testing more streamlined and less expensive — and potentially more accessible to millions of people with rare diseases.

Rare Disease Day at NIH: Drawing Hope and Building Connections Across the Community

May 1, 2020 - NCATS News

  • Genetic and Rare Diseases (GARD) Information Center

The annual event — held this year on February 28, 2020 — focused on forging connections in the rare diseases community, shortening the journey toward diagnosis and pursuing personalized medicine as a therapeutic approach for treating rare diseases.

Last updated on September 8, 2025