FDA orphan drug designations for lysosomal storage disorders - a cross sectional analysis
Sven F. Garbade, Matthias Zielonka, Konstantin Mechler, Stefan Kölker, Georg Friedrich Hoffmann, Christian Staufner, Eugen Mengel, Markus Ries
The paper analyzes FDA orphan drug designations for lysosomal storage disorders, showing growth in approved therapies and diversification into small molecules and gene therapy.
PLoS ONE · Apr 8, 2020Peer-reviewedGenetic & Genomic Medicine