发明名称 |
METHODS AND COMPOSITIONS FOR CNS DELIVERY OF ß-GALACTOCEREBROSIDASE |
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 B-Galactocerebrosidase protein, salt, and a polysorbate surfactant for the treatment of GLD Disease. |
申请公布号 |
WO2011163651(A2) |
申请公布日期 |
2011.12.29 |
申请号 |
WO2011US41927 |
申请日期 |
2011.06.25 |
申请人 |
SHIRE HUMAN GENETIC THERAPIES, INC.;SALAMAT-MILLER, NAZILA;TAYLOR, KATHERINE;MANNING, KEN;ZHU, GAOZHONG |
发明人 |
SALAMAT-MILLER, NAZILA;TAYLOR, KATHERINE;MANNING, KEN;ZHU, GAOZHONG |
分类号 |
A61K38/47;A61P25/00;A61P25/28 |
主分类号 |
A61K38/47 |
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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