Article

FDA orphan drug designations for lysosomal storage disorders – a cross sectional analysis

2020-01-10

Abstract excerpt

<h4>Purpose</h4> To provide a quantitative clinical-regulatory insight into the status of FDA orphan drug designations for compounds intended to treat lysosomal storage disorders (LSD’s). <h4>Methods</h4> Assessment of the drug pipeline through analysis of the FDA database for orphan drug designations with descriptive and comparative statistics. <h4>Results</h4> Between 1983 and 2019, 124 orphan drug designations...

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Literature Corpus work: 882f02fb-80d4-5d35-86d6-b8266607b09f
DOI: 10.1101/2020.01.05.20016568

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