P189: Vestronidase alfa for the treatment of mucopolysaccharidosis VII (MPS VII): Updated results from a novel, longitudinal, multicenter disease monitoring program

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Bibliographic Details
Published in:Genetics in Medicine Open 2023, Vol.1 (1), p.100218, Article 100218
Main Authors: Giugliani, Roberto, Gonzalez-Meneses, Antonio, Grant, Christina, Scarpa, Maurizio, Sun, Angela, Wang, Raymond, Hetzer, Joel, Marsden, Deborah, Merritt, J. Lawrence
Format: Article
Language:English
Online Access:Get full text
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ISSN:2949-7744
2949-7744
DOI:10.1016/j.gimo.2023.100218