Efficient and Innovative Natural History Studies Addressing Unmet Needs in Rare Diseases (R01 Clinical Trials Optional)
This NOFO supports efficient, innovative natural history studies in rare diseases where there are major unmet medical and development needs. The goal is to generate high-quality, interpretable data that fills knowledge gaps, reduces barriers, and helps advance medical product development for one or more rare diseases with related biology. Clinical trials are optional under this R01 mechanism.<…
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