Genzyme Coorporation;Icahn School of Medicine at Mount Sinai
发明人:
SCHUCHMAN Edward H.,DESNICK Robert J.,COX Gerald F.,ANDREWS Laura P.,MURRAY James M.
申请号：
SI201031915
公开号：
SI2470199T1
申请日:
2010.08.28
申请国别(地区):
SI
年份:
2019
代理人:
摘要：
The invention relates to recombinant human acid sphingomyelinase (rhASM) for use in a method of treating an acid sphingomyelinase deficiency (ASMD) such as, for example, Niemann Pick Disease type B or non-neuronopathic ASMD in a human subject, comprising a dose escalation regimen comprising administering to the human subject one or more initial doses of rhASM, wherein the initial dose ranges between 0.001 mg/kg and 0.1 mg/kg rhASM (for example, the initial dose is 0.03 mg/kg rhASM or 0.1 mg/kg rhASM) and administering successively higher doses of rhASM (for example, 0.1 mg/kg, 0.3 mg/kg, 0.6 mg/kg, and 1 mg/kg rhASM) to the human subject, if the subject does not manifest one or more moderate or severe adverse events that has/have a causal relationship with the treatment, wherein the successively higher doses are from 0.1 mg/kg to 1.0 mg/kg rhASM higher than the previous dose, or 0.1 mg/kg to 0.5 mg/kg rhASM higher than the previous dose.
