Clinical Trials for New Indications in Rare Neurodegenerative Diseases (R01)
This R01 funds clinical trials testing the safety and/or efficacy of products to support new indications or labeling changes that address unmet needs in rare neurodegenerative diseases for children and adults. It prioritizes collaborative, efficient, and innovative trial designs to accelerate FDA approval of treatments and advance rare disease drug development. Funded studies should directly s…
The purpose is to fund clinical trials of products evaluating efficacy and/or safety in support of a new indication or change in labeling to address unmet needs in rare neurodegenerative diseases for children and adults. Through the support of collaborative, efficient, and/or innovative clinical…
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