Preclinical Proof of Concept Studies for Rare Diseases (R21 Clinical Trial Not Allowed)
NCATS invites R21 applications to run rigorous efficacy studies of a potential therapeutic in an established, validated rare-disease preclinical model, showing proof of concept for further development. Projects should include pharmacokinetic and pharmacodynamic measures alongside efficacy endpoints, and may support additional PK if feasible. Applicants must include a plan for a rare-disease st…
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