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ENZYME SUBSTITUTION THERAPY WITH INCREASED DOSE FOR TREATMENT OF ACID Sphingomyelinase deficiency
ENZYME SUBSTITUTION THERAPY WITH INCREASED DOSE FOR TREATMENT OF ACID Sphingomyelinase deficiency
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机译：剂量增加的酶替代疗法治疗酸性鞘磷脂酶缺乏症
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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.
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机译：本发明涉及重组人酸性鞘磷脂酶（rhASM），其用于在人受试者中治疗酸性鞘磷脂酶缺乏症（ASMD）例如Niemann Pick Disease B型或非神经病变性ASMD的方法，其包括剂量递增方案，其包括向人受试者施用一或多个初始剂量的rhASM，其中初始剂量的范围为0.001 mg / kg至0.1 mg / kg rhASM（例如，初始剂量为0.03 mg / kg rhASM或0.1 mg / kg rhASM ），并且如果受试者未表现出一种或多种中度或高剂量，则连续向人受试者施用更高剂量的rhASM（例如0.1 mg / kg，0.3 mg / kg，0.6 mg / kg和1 mg / kg rhASM）。与治疗有因果关系的严重不良事件，其中相继较高的剂量比先前的剂量高0.1 mg / kg至1.0 mg / kg rhASM，或比先前的剂量高0.1 mg / kg至0.5 mg / kg rhASM以前的剂量。
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