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.
Transparency and Early Planning Are Key in Increasing Efficiency of Gene Therapy Development for Rare Diseases
February 26, 2026 - NCATS News
- Platform Vector Gene Therapy (PaVe-GT)
PaVe-GT scientists underscore strategy, framework and early interaction with the FDA to align preclinical and clinical efforts in AAV gene therapy translation.
Read ArticleCures Within Reach Selected Three Clinical Trials in Rare/Ultra-Rare Diseases to Validate AI Predictions
February 25, 2026 - Grantee/Partner News
- Biomedical Data Translator (Translator)
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.
Clinical Trial Readiness Grants Aim to Set the Stage for Rare Disease Clinical Trials
March 17, 2020 - NCATS News
- Genetic and Rare Diseases (GARD) Information Center
To close the gap between development of candidate interventions for rare diseases and clinical testing, NCATS and NICHD created rare-disease Clinical Trial Readiness grants. Funds support projects that collect data needed to move on to clinical trials.
Collaborative Approach Aids Development of Potential Blood Test for Use in Alzheimer’s, Rare Syndromes Diagnoses
March 2, 2020 - NCATS News
- Genetic and Rare Diseases (GARD) Information Center
Contributions from the NCATS-led Rare Diseases Clinical Research Network helped develop a new blood test that may make it easier to diagnose Alzheimer’s disease and rare neurodegenerative syndromes.