Successfully Navigating FDA Orphan Drug and Rare Pediatric Disease Designations for AAV9-hPCCA Gene Therapy: The NIH Pave-GT Experience.
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DOI: 10.1089/hum.2022.232
Abstract: Orphan drug designation (ODD) is an important program intended to facilitate the development of orphan drugs in the US. An orphan drug benefitting pediatric patients can qualify as a drug for a Rare Pediatric Disease… read more here.
Keywords: orphan drug; gene; rpdd; gene therapy ... See more keywords