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New Funding Opportunities Available for Rare Diseases Research

NCATS has released two new funding opportunities to support research on rare diseases through the Rare Diseases Clinical Research Network.


Potential Gene-Editing Treatment Advances to Clinical Trials

RDCRN-supported researchers charted how several rare diseases progressed in patients over time, helping to enable a potential gene-editing therapy to advance to clinical trials.

Christopher P. Austin, M.D., Anne R. Pariser, M.D., Ronald J. Bartek, Petra Kaufmann, M.D., M.Sc., and Annie Kennedy

NCATS Unveils Patient-Focused Therapy Development Toolkit

NCATS showcases web-based platform of patient-focused rare diseases research resources at NIH event in September.

Have a rare disease?

The Genetic and Rare Diseases Information Center (GARD) offers credible, up-to-date health information for many rare diseases.

Interested in therapy development?

Access tools that can help patient groups advance medical research and patient-focused therapy development via the NCATS Toolkit for Patient-Focused Therapy Development.

Patient/Community Engagement & Health Information

To ensure patient involvement and community engagement throughout the research process, institutions must collaborate with community organizations and patient groups to identify and understand public health needs.

Find NCATS Programs & InitiativesAccess NCATS Expertise & Resources

Genetic & Rare Diseases

The Genetic and Rare Diseases Information Center provides up-to-date health information about numerous rare and genetic diseases.

Patient-Focused Therapy Development

The NCATS Toolkit for Patient-Focused Therapy Development is a collection of online resources that can help patient groups advance through the process of therapy development.

Undiagnosed Diseases

The goals of the Undiagnosed Diseases Program, led by the NIH Common Fund, are to provide answers and advance medical knowledge about rare and common diseases.

Clinical Trials at NIH

Patient involvement in clinical research helps investigators uncover better ways to treat, prevent, diagnose and understand human diseases.

Principles of Community Engagement

This comprehensive guide, available in English and Spanish (PDF - 3.29MB), outlines core principles for engaging diverse communities in clinical research activities.

Therapy Development Resources

The NCATS Toolkit for Patient-Focused Therapy Development (Toolkit) was created to provide a collection of online resources that can help patient groups advance through the process of therapy development and provide them with the tools they need to advance medical research. Download the Toolkit fact sheet (PDF - 645KB).