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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Dades bibliogràfiques
Publicat a:PLoS One
Autors principals: Garbade, Sven F., Zielonka, Matthias, Mechler, Konstantin, Kölker, Stefan, Hoffmann, Georg F., Staufner, Christian, Mengel, Eugen, Ries, Markus
Format: Artigo
Idioma:Inglês
Publicat: Public Library of Science 2020
Matèries:
Research Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC7141691/
https://ncbi.nlm.nih.gov/pubmed/32267884
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0230898
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