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. |
申请人 |
SHIRE HUMAN GENETIC THERAPIES, INC. |
发明人 |
ZHU, GAOZHONG;LOWE, KRIS;SHAHROKH, ZAHRA;CHRISTIAN, JAMES;FAHRNER, RICK;PAN, JING;WRIGHT, TERESA, LEAH;CALIAS, PERICLES |