Successfully Navigating Food and Drug Administration Orphan Drug and Rare Pediatric Disease Designations for AAV9-hPCCA Gene Therapy: The National Institutes of Health Platform Vector Gene Therapy Experience

Orphan drug designation (ODD) is an important program intended to facilitate the development of orphan drugs in the United States. An orphan drug benefiting pediatric patients can qualify as a drug for a Rare Pediatric Disease Designation (RPDD) as well. The ODD and RPDD programs provide financial i...

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Detalles Bibliográficos
Autores principales: Lomash, Richa Madan, Shchelochkov, Oleg, Chandler, Randy J., Venditti, Charles P., Pariser, Anne R., Ottinger, Elizabeth A.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Mary Ann Liebert, Inc., publishers 2023
Materias:
Clinical Developments
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10031144/
https://www.ncbi.nlm.nih.gov/pubmed/36694456
http://dx.doi.org/10.1089/hum.2022.232

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