METHODS AND COMPOSITIONS FOR CNS DELIVERY OF HEPARAN N-SULFATASE

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专利权人:

Inc.;Shire Human Genetic Therapies

发明人:

Natoli, Farah,Zhu, Gaozhong,Terew, Jennifer,Jiang, Yuan,Tsung, Jamie,Shahrokh, Zahra,Vernaglia, Brian,Pan, Jing,Pfeifer, Richard,Calias, Pericles

申请号：

AU2017202216

公开号：

AU2017202216A1

申请日:

2017.04.04

申请国别(地区):

AU

年份:

2017

代理人:

摘要：

#$%^&*AU2017202216A120170427.pdf#####ABSTRACT 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 a heparan N-sulfatase (HNS) protein, salt, and a polysorbate surfactant for the treatment of Sanfilippo Syndrome Type A.

来源网站：

中国工程科技知识中心

来源网址：

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