Disease characteristics, early effectiveness, and safety of vestronidase alfa for the treatment of mucopolysaccharidosis type VII (MPS VII) assessed in a novel, longitudinal, multicenter Disease Monitoring Program (DMP)

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Bibliographic Details
Published in:Molecular genetics and metabolism 2021-02, Vol.132 (2), p.S63-S63
Main Authors: Lau, Heather, Gonzalez-Meneses, Antonio, Scarpa, Maurizio, Hostutler, Robert, Zhang, Lin, Malkus, Betsy, Ramirez, Antonio Nino, Marsden, Deborah, Giugliani, Roberto
Format: Article
Language:English
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ISSN:1096-7192
1096-7206
DOI:10.1016/j.ymgme.2020.12.141