文章基本信息

标题：Patient's weight can decide about spending millions on enzyme replacement therapy in MPS II
作者：Miroslaw Bik-Multanowski ; Ewa Jamroz ; Lukasz Kaluzny 等
期刊名称：Molecular Genetics and Metabolism Reports
印刷版ISSN：2214-4269
出版年度：2016
卷号：6
页码：5-7
DOI：10.1016/j.ymgmr.2015.12.002
出版社：Elsevier B.V.
关键词：Pediatric orphan disease; Enzyme replacement; Public health; Expensive therapies; Walking test