ZHU, Gaozhong,LOWE, Kris,SHAHROKH, Zahra,PAN, Jing,WRIGHT, Teresa, Leah,CALIAS, Pericles,CHRISTIAN, James,FAHRNER, Rick
申请号：
DK11799036
公开号：
DK2593131T3
申请日:
2011.06.25
申请国别(地区):
DK
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.
