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A comprehensive study of the rare diseases and conditions targeted by orphan drug designations and approvals over the forty years of the Orphan Drug Act

BACKGROUND: Rare diseases affect more than 30 million Americans. The passage of the Orphan Drug Act (ODA) in the United States in 1983 represented a launching point for a rare disease drug development revolution for these patients. Financial incentives provided by the ODA through its Orphan Drug Des...

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Detalles Bibliográficos
Autores principales: Fermaglich, Lewis J., Miller, Kathleen L.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10290406/
https://www.ncbi.nlm.nih.gov/pubmed/37353796
http://dx.doi.org/10.1186/s13023-023-02790-7