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To provide a quantitative clinical-regulatory insight into the status of FDA orphan drug designations for compounds intended to treat lysosomal storage disorders (LSDs). Assessment of the drug pipeline through analysis of the FDA database for orphan drug designations with descriptive and comparative statistics. Between 1983 and 2019, 124 orphan drug designations were granted by the FDA for compounds intended to treat 28 lysosomal storage diseases. Orphan drug designations focused on Gaucherdoi:10.1371/journal.pone.0230898 pmid:32267884 fatcat:kw44yeqvwbecfcprvcu3vhmasu