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 descriptive...
| Main Authors: | , , , , , , , |
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| Format: | Article |
| Language: | English |
| Published: |
Public Library of Science (PLoS)
2020-01-01
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| Series: | PLoS ONE |
| Online Access: | https://doi.org/10.1371/journal.pone.0230898 |