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supports natural history studies that improve medical product development for rare diseases with unmet needs. The goal is to fill knowledge gaps, remove barriers, and help advance treatments for one or more rare diseases with similar causes.
Full Description
the purpose of this notice of funding opportunity (nofo) is to support efficient and innovative natural history studies that advance medical product development in rare diseases/conditions with unmet needs. through the support of natural history studies with high quality and interpretable data elements, fda expects to address critical knowledge gaps, remove major barriers to progress in the field, exert a significant and broad impact on a specific rare disease or multiple rare diseases with similar pathophysiology, and facilitate rare disease product development.
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