Mount Sinai School of Medicine of New York University;Genzyme Corporation
发明人:
Schuchman, Edward H.,Desnick, Robert J.,Cox, Gerald F.,Andrews, Laura P.,Murray, James M.
申请号：
AU2010286550
公开号：
AU2010286550A1
申请日:
2010.08.28
申请国别(地区):
AU
年份:
2012
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.
