Funds clinical trials for new treatments or labeling changes for rare neurodegenerative diseases in adults and children, aiming to increase FDA-approved therapies.
This grant funds innovative natural history studies to improve understanding and support medical product development for rare diseases with unmet needs.
This grant funds innovative natural history studies to advance medical product development and address unmet needs in rare diseases by generating high-quality, interpretable data.
Funding available for preclinical studies in rare diseases to show effectiveness of proposed treatments for potential further development into clinical trials.
This grant will fund research to improve clinical trial readiness for rare diseases by developing tools like biomarkers and outcome measures to help move new treatments or diagnostics toward successful trials.