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.
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.
This grant funds innovative natural history studies to improve understanding and support medical product development for rare diseases with unmet needs.
Funds clinical trials testing new or existing treatments to improve care and expand approved uses for rare diseases with unmet medical needs.
Encourages translational activities and small clinical studies for neural devices to address disorders affecting the nervous system; includes prototype development, safety testing, and clinical validation.