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
Introduction 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 orp...
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| Hauptverfasser: | , , , , |
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| Dokumenttyp: | Article (Journal) Kapitel/Artikel |
| Sprache: | Englisch |
| Veröffentlicht: |
18 May 2022
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| Ausgabe: | Version 1 |
| In: |
Research Square
Year: 2022, Pages: 1-13 |
| ISSN: | 2693-5015 |
| DOI: | 10.21203/rs.3.rs-1639622/v1 |
| Online-Zugang: | Resolving-System, kostenfrei, Volltext: https://doi.org/10.21203/rs.3.rs-1639622/v1 Verlag, kostenfrei, Volltext: https://www.researchsquare.com 
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| Verfasserangaben: | Franziska C Trudzinski, Maria Ada Presotto, Emanuel Buck, Felix J. Herth, Markus Ries |
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| 520 | |a Introduction 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 orp... | ||
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