First human treatment with investigational rhGUS enzyme replacement therapy in an advanced stage MPS VII patient

标题
First human treatment with investigational rhGUS enzyme replacement therapy in an advanced stage MPS VII patient
作者
关键词
Mucopolysaccharidosis type VII, Enzyme replacement therapy, Pulmonary function, Physician global impression of change, Hepatosplenomegaly, Sly syndrome
出版物
MOLECULAR GENETICS AND METABOLISM
Volume 114, Issue 2, Pages 203-208
出版商
Elsevier BV
发表日期
2014-11-08
DOI
10.1016/j.ymgme.2014.10.017

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