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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 descripti...
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| Publicado no: | PLoS One |
|---|---|
| Main Authors: | , , , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Public Library of Science
2020
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7141691/ https://ncbi.nlm.nih.gov/pubmed/32267884 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0230898 |
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