NCATS is committed to using research to address the public health crisis presented by rare diseases. Speeding development of treatments for patients requires innovation in science and technology and engaging patients and their support organizations as essential partners.
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Research
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- Funding & Notices
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Rare Diseases Research and Resources
Register: New Challenge Aims to Advance Genome Editing Delivery Technology
The TARGETED (Targeted Genome Editor Delivery) Challenge aims to advance genome editing delivery technology. The Challenge is sourcing creative ways to deliver genome editors to somatic cells.
Rare Diseases Clinical Research Network (RDCRN)The RDCRN program is designed to advance medical research on rare diseases by facilitating collaboration, study enrollment and data sharing.
Gene Therapy and Gene Editing ProgramsNCATS is involved with multiple programs for accelerating gene-targeted therapies using approaches that speed the development of treatments for multiple rare diseases at a time.
Therapeutics for Rare and Neglected Diseases (TRND)The TRND program supports preclinical development of therapeutic candidates intended to treat rare or neglected disorders, with the goal of enabling an Investigational New Drug application.
Resources for People with Rare DiseasesTo ensure patient involvement and engagement throughout the research process, institutions must collaborate with patients and patient groups to identify and understand public health needs.
Additional Rare Diseases Research and InitiativesNCATS funds extramural research and participates in initiatives and activities aimed at addressing rare diseases.
Rare Diseases Clinical Research Network (RDCRN)
The RDCRN program is designed to advance medical research on rare diseases by facilitating collaboration, study enrollment and data sharing.
Gene Therapy and Gene Editing Programs
NCATS is involved with multiple programs for accelerating gene-targeted therapies using approaches that speed the development of treatments for multiple rare diseases at a time.
Therapeutics for Rare and Neglected Diseases (TRND)
The TRND program supports preclinical development of therapeutic candidates intended to treat rare or neglected disorders, with the goal of enabling an Investigational New Drug application.
Resources for People with Rare Diseases
To ensure patient involvement and engagement throughout the research process, institutions must collaborate with patients and patient groups to identify and understand public health needs.
Additional Rare Diseases Research and Initiatives
NCATS funds extramural research and participates in initiatives and activities aimed at addressing rare diseases.
Cost of Living with a Rare Disease
An NCATS-led study finds that the number of individuals with rare diseases and their medical costs have been underestimated, suggesting that nationwide health care costs associated with rare diseases are likely similar to common diseases such as cancer.
Learn more about the IDeaS (Impact of Rare Diseases on Patients and Healthcare Systems) study.
Rare Diseases Research Funding
NCATS seeks to facilitate rare diseases research by enabling efficient and effective movement of candidate therapies and diagnostics toward clinical trials and to increase their likelihood of success.
Learn more about Clinical Trial Readiness for Rare Diseases, Disorders and Syndromes grants, including current funding opportunities.
Browse our Open Funding Opportunities for additional rare diseases research funding.
Interested in Clinical Trials at NIH?
Patient involvement in clinical research helps investigators uncover better ways to treat, prevent, diagnose and understand human diseases.
Visit ClinicalTrials.gov for information about clinical studies at NIH and other institutions that are currently enrolling patients.
Division of Rare Diseases Research Innovation
Get in touch with Division of Rare Diseases Research Innovation staff for more information on programs and areas of expertise.
Stay connected with the Division of Rare Diseases Research Innovation on Facebook and Twitter.
- Funding & Notices
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