שיטות ותכשירים למתן של אידורונאט–2–סולפטאז למערכת העצבים המרכזית

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专利权人:

SHIRE HUMAN GENETIC THERAPIES INC.

发明人:

申请号：

IL26808519

公开号：

IL268085D0

申请日:

2019.07.15

申请国别(地区):

IL

年份:

2019

代理人:

摘要：

The present invention provides, among other things, compositions and methods for CNS delivery of lysosomal enzymes for effective treatment of lysosomal storage diseases. In some embodiments, the present invention includes a stable formulation for direct CNS intrathecal administration comprising an iduronate-2-sulfatase (I2S) protein, salt, and a polysorbate surfactant for the treatment of Hunters Syndrome.

来源网站：

中国工程科技知识中心

来源网址：

http://www.ckcest.cn/home/