Robert J. Desnick;Laura P. Andrews;James M. Murray;Gerald F. Cox;Edward H. Schuchman
发明人:
Edward H. Schuchman,Robert J. Desnick,Gerald F. Cox,Laura P. Andrews,James M. Murray
申请号：
US12870790
公开号：
US08349319B2
申请日:
2010.08.28
申请国别(地区):
US
年份:
2013
代理人:
摘要：
The invention relates to dose escalation enzyme replacement therapy using acid sphingomyelinase (ASM) for the treatment of human subjects having acid sphingomyelinase deficiency (ASMD), and, in particular, patients with non-neurological manifestations of Niemann-Pick Disease (NPD), and in certain embodiments, NPD type B.
