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.
Funds clinical trials testing new or existing treatments to improve care and expand approved uses for rare diseases with unmet medical needs.
Funding available for preclinical studies in rare diseases to show effectiveness of proposed treatments for potential further development into clinical trials.
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 early phase clinical trials testing natural products like botanicals, probiotics, and dietary supplements with strong scientific backing for further testing.