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The outcome of more than 3 decades of orphan drug development in alpha-1-antitypsin deficiency – a cross sectional analysis of the FDA Orphan Drug Product designation database

2022-05-18

Abstract excerpt

<h4>Introduction: </h4> Alpha-1 antitrypsin deficiency (AATD, OMIM #613490) is a rare metabolic disorder affecting lungs and liver. The purpose of this study is to assess the impact of the US orphan drug act on AATD by providing a quantitative clinical-regulatory insight into the status of FDA orphan drug approvals and designations for compounds intended to treat AATD. <h4>Methods:</h4> Cross-sectional analysis of...

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Literature Corpus work
a16f8ed8-3b27-5a1b-ad6e-2546a5a2f309
DOI
10.21203/rs.3.rs-1639622/v1
Open publication

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