КАЛИАС Периклс (US),ПАН Цзинь (US),ПАУВЕЛЛ Джен (US),ЧАРНАС Лоуренс (US),МАККОЛИ Томас (US),РАЙТ Тереса Леах (US),ПФАЙФЕР Ричард (US),ШАХРОКХ Захра (US)
申请号：
RU2012154831/15
公开号：
RU2012154831A
申请日:
2011.06.25
申请国别(地区):
RU
年份:
2014
代理人:
摘要：
1. A method comprising the step of: intrathecal administration to a subject suffering from or predisposed to lysosomal storage disease associated with a decrease in the level or activity of the lysosomal enzyme, a composition containing a replacement enzyme for replacing the lysosomal enzyme at a concentration above about 5 mg / ml. The method according to claim 1, characterized in that the concentration of the replacement enzyme is above about 10 mg / ml. The method according to claim 1, characterized in that the composition further comprises one or more of (I) a buffering agent, (II) a surfactant, or (III) a tonicity regulator. The method according to any one of claims 1 to 3, characterized in that the pH of the composition is about 3.0-8.0.5. The method according to any one of claims 1 to 3, characterized in that the composition is administered in a single dose of less than 5 ml. A method according to any one of claims 1 to 3, characterized in that the composition is administered in a single dose of less than 3 ml. A method according to any one of claims 1 to 3, characterized in that the intrathecal administration of the composition does not cause significant side effects in the subject. A method according to any one of claims 1 to 3, characterized in that the intrathecal administration of the composition does not lead to an adaptive T-cell immune response. A method comprising the step of: intrathecal administration to a subject suffering from or predisposed to lysosomal storage disease associated with a decrease in the level or activity of the lysosomal enzyme, a composition containing a replacement enzyme for replacing the lysosomal enzyme, in a composition that is not a synthetic CSF. 10. The method according to claim 9, characterized in that the concentration of the replacement enzyme is above about 5 mg / ml. The method comprising the stage of:1. Способ, включающий стадию:интратекального введения субъекту, страдающему от или предрасположенного к лизосомной б
