SALAMAT-MILLER, Nazila N,TAYLOR, Katherine K,CAMPOLIETO, Paul P,SHAHROKH, Zahra Z,PAN, Jing J,CHARNAS, Lawrence L,WRIGHT, Teresa Leah TL,CALIAS, Pericles P,.薩拉馬特.米勒,.泰勒,.坎波列托,.夏若克,.潘,.恰爾納斯,.萊特,.加萊斯
申请号：
HK16102145.2
公开号：
HK1214149A
申请日:
2016.02.25
申请国别(地区):
HK
年份:
2016
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.
