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Resources for Rare Disease Patients and Advocates

We support and provide materials and information about rare diseases. Explore our rare diseases resources to find ways to get involved and learn about the latest research.

Genetic and Rare Diseases (GARD) Information Center

The GARD Information Center provides current and reliable health information on a wide range of genetic and rare diseases.

NCATS Toolkit for Patient-Focused Therapy Development

Our Toolkit for Patient-Focused Therapy Development is a collection of online resources to assist patient groups in the research and development of new treatments.

Facts and Stats

Explore Community Resources

We provide a range of free materials and resources to help patients, caregivers, patient support organizations, health care providers and scientists learn about rare diseases and advance research in this area.

Rare Diseases Registry Program (RaDaR)

This resource provides best practices for setting up and maintaining patient registries for research purposes.

Rare Disease Day at NIH

Rare Disease Day at NIH aims to raise awareness about rare diseases, the people they affect, and NIH collaborations that address scientific challenges and advance research for new treatments.

Rare Disease News

Our latest news stories showcase the rare diseases research we conduct and support.

Learn About Rare Diseases Research at NCATS

We support research programs and engage in activities to address the public health challenges of rare diseases.

Gene-Targeted Therapy Programs

We support and participate in multiple programs that speed the development of gene-targeted therapies for many diseases at a time.

Therapeutics for Rare and Neglected Diseases (TRND)

Through this in-house program, we support preclinical development of therapeutic options for diseases with high unmet medical needs.

Rare Diseases Clinical Research Network (RDCRN)

We oversee this NIH-wide program that supports medical research on over 200 rare diseases through clinical studies, collaborations, study enrollment and data sharing. 

Last updated on May 13, 2024