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Orphan drug development in alpha-1 antitypsin deficiency

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 des...

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Detalles Bibliográficos
Autores principales:	Trudzinski, Franziska C., Presotto, Maria Ada, Buck, Emanuel, Herth, Felix J. F., Ries, Markus
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Nature Publishing Group UK 2022
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9477815/ https://www.ncbi.nlm.nih.gov/pubmed/36109566 http://dx.doi.org/10.1038/s41598-022-19707-2

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