PROCÉDÉS ET COMPOSITIONS POUR UNE ADMINISTRATION AU SNC D'HÉPARANE 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：

EP11799034

公开号：

EP2588131A4

申请日:

2011.06.25

申请国别(地区):

EP

年份:

2014

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 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/