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US 11,998,592 B2Granted
Methods of treating acid sphingomyelinase deficiency
AssigneeGenzyme Corporation
InventorsWei-Lien Chuang, Gerald F. Cox, X. Kate Zhang
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Abstract
The present disclosure provides methods of screening, diagnosing, monitoring and/or treating acid sphingomyelinase (ASM) disorders such as Niemann-Pick disease. In particular, the methods encompass techniques for improved diagnosis and/or treatment of an ASM disorder, for example using enzyme replacement therapy.
Claims5
A method of treating acid sphingomyelinase deficiency in a human subject, wherein the method comprises:
- administering to the subject intravenously a starting dose of recombinant human acid sphingomyelinase (rhASM) or a modified rhASM at 0.03 or 0.1 mg/kg and subsequent doses that increase in dose concentration until reaching 3 mg/kg;
- administering maintenance doses to the subject at 3 mg/kg or less;
- and
- measuring the level of lyso-sphingomyelin (lyso-SPM) in a biological sample collected from the subject (a) within 24 hours, (b) within 48 hours, or (c) 72 or more hours after administration of a dose;
- wherein each dose is administered two weeks after the previous dose.
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Dependencies are inferred from the claim text.