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Investigational intrathecal (IT) enzyme replacement therapy for the severe form of Hunter syndrome (mucopolysaccharidosis type II, MPS II)

Joseph Muenzer,Christian J Hendriksz,Margot B Stein,Zheng Fan,Shauna Kearney,J C Horton,Suresh Vijayaraghavan,V H Perry,Saikat Santra,Guirish A Solanki,Nan Wang,Mary Ann Mascelli,Luying Pan,Kenneth Sciarappa,Ann J Barbier

MOLECULAR GENETICS AND METABOLISM（2014）

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Key words
mucopolysaccharidosis type ii,hunter syndrome,enzyme replacement therapy
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