Preclinical Proof of Concept Studies for Rare Diseases (R21 Clinical Trial Not Allowed)
This funding opportunity supports preclinical proof-of-concept efficacy studies for therapies targeting rare diseases using established preclinical models. Applicants can also include pharmacodynamic and pharmacokinetic work to show the agent’s therapeutic activity and biological effects, without conducting any clinical trials. The aim is to generate evidence that the therapeutic is ready for …
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