Preliminary clinical efficacy and safety of BMN 701, GILT-tagged recombinant human acid alpha glucosidase (rhGAA), in late-onset Pompe disease: results of an extension study

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Preliminary clinical efficacy and safety of BMN 701, GILT-tagged recombinant human acid alpha glucosidase (rhGAA), in late-onset Pompe disease: results of an extension study

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作者：Barry Byrne ; Richard Barohn ; Bruce Barshop ; Drago Bratkovic ; Claude Desnuelle ; Robert Henderson ; Tarekegn Hiwot ; Derralynn Hughes ; Pascal Laforet ; Eugen Mengel ; Mark Roberts ; William Lang ; Jonathon LeBowitz
刊名：Molecular Genetics and Metabolism
出版年：February, 2014
年：2014
卷：111
期：2
页码：S29
全文大小：49 K

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