SHAHROKH ZAHRA,CALIAS PERICLES,PAN JING,POWELL JAN,CHARNAS LAWRENCE,MCCAULEY THOMAS,WRIGHT TERESA LEAH,PFEIFER RICHARD
申请号：
NZ60586511
公开号：
NZ605865A
申请日:
2011.06.25
申请国别(地区):
NZ
年份:
2015
代理人:
摘要：
Disclosed is the use of a composition comprising a replacement enzyme in the manufacture of a medicament for treating a lysosomal storage disease associated with reduced level or activity of a lysosomal enzyme, wherein the replacement enzyme is present in the composition at a concentration at or greater than about 5 mg/ml, the composition comprises no greater than 50 mM of phosphate, and the treating comprises a step of administering the composition intrathecally to a subject in need of treatment.
