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

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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 de...

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Main Authors: Garbade, Sven (Author) , Zielonka, Matthias (Author) , Mechler, Konstantin (Author) , Kölker, Stefan (Author) , Hoffmann, Georg F. (Author) , Staufner, Christian (Author) , Mengel, Eugen (Author) , Ries, Markus (Author)
Format: Article (Journal)
Language:English
Published: April 8, 2020
In: PLOS ONE
Year: 2020, Volume: 15, Issue: 4
ISSN:1932-6203
DOI:10.1371/journal.pone.0230898
Online Access:Verlag, lizenzpflichtig, Volltext: https://doi.org/10.1371/journal.pone.0230898
Verlag, lizenzpflichtig, Volltext: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7141691/
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Author Notes:Sven F. Garbade, Matthias Zielonka, Konstantin Mechler, Stefan Kölker, Georg F. Hoffmann, Christian Staufner, Eugen Mengel, Markus Ries
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FDA orphan drug designations for lysosomal storage disorders: a cross sectional analysis by Garbade, Sven (Author) , Zielonka, Matthias (Author) , Mechler, Konstantin (Author) , Kölker, Stefan (Author) , Hoffmann, Georg F. (Author) , Staufner, Christian (Author) , Mengel, Eugen (Author) , Ries, Markus (Author) ,


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