发明名称 |
METHODS AND COMPOSITIONS FOR CNS DELIVERY OF HEPARAN N-SULFATASE |
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. |
申请公布号 |
US2012014936(A1) |
申请公布日期 |
2012.01.19 |
申请号 |
US201113168957 |
申请日期 |
2011.06.25 |
申请人 |
NATOLI FARAH;ZHU GAOZHONG;TEREW JENNIFER;JIANG YUAN;TSUNG JAMIE;SHAHROKH ZAHRA;VERNAGLIA BRIAN;PAN JING;PFEIFER RICHARD;CALIAS PERICLES;SHIRE HUMAN GENETIC THERAPIES, INC. |
发明人 |
NATOLI FARAH;ZHU GAOZHONG;TEREW JENNIFER;JIANG YUAN;TSUNG JAMIE;SHAHROKH ZAHRA;VERNAGLIA BRIAN;PAN JING;PFEIFER RICHARD;CALIAS PERICLES |
分类号 |
A61K38/46;A61P25/00;A61P27/16;A61P43/00 |
主分类号 |
A61K38/46 |
代理机构 |
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代理人 |
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主权项 |
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地址 |
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