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FDA orphan drug designations for lysosomal storage disorders – a cross-sectional analysis

PURPOSE: To provide a quantitative clinical-regulatory insight into the status of FDA orphan drug designations for compounds intended to treat lysosomal storage disorders (LSDs). METHODS: Assessment of the drug pipeline through analysis of the FDA database for orphan drug designations with descripti...

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Detalles Bibliográficos
Autores principales:	Garbade, Sven F., Zielonka, Matthias, Mechler, Konstantin, Kölker, Stefan, Hoffmann, Georg F., Staufner, Christian, Mengel, Eugen, Ries, Markus
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Public Library of Science 2020
Materias:	Research Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7141691/ https://www.ncbi.nlm.nih.gov/pubmed/32267884 http://dx.doi.org/10.1371/journal.pone.0230898

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