Genetic & Genomic MedicinePLoS ONEPeer-reviewed
FDA orphan drug designations for lysosomal storage disorders - a cross sectional analysis
Sven F. Garbade, Matthias Zielonka, Konstantin Mechler…·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.
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