Iduronate-2-Sulfatase with Anti-human Transferrin Receptor Antibody for Neuropathic Mucopolysaccharidosis II: A Phase 1/2 Trial

出版年份 2018 全文链接

标题

作者

关键词

mucopolysaccharidosis II, Hunter syndrome, blood brain barrier, iduronate-2-sulfatase, CNS, cognitive impairment, anti-human transferrin receptor antibody, heparan sulfate, dermatan sulfate, enzyme-replacement therapy, clinical trial

出版物

MOLECULAR THERAPY

Volume -, Issue -, Pages -

出版商

Elsevier BV

发表日期

2018-12-08

DOI

10.1016/j.ymthe.2018.12.005

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