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 clinical trials to test the safety and effectiveness of treatments for rare diseases, aiming to address unmet needs and support new or updated drug approvals.
Funds clinical trials testing new or existing treatments to improve care and expand approved uses for rare diseases with unmet medical 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.