Genzyme Corporation;Icahn School of Medicine at Mount Sinai
发明人:
Edward H. Schuchman,Robert J. Desnick,Gerald F. Cox,Laura P. Andrews,James M. Murray
申请号：
US14156894
公开号：
US20140335070A1
申请日:
2014.01.16
申请国别(地区):
US
年份:
2014
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.
