Patent ID: 9655954
Date: 2017-05-23
CPC Classifications: A61K,A61P,C12Y

Claim:
1. A method for reducing sphingomyelin accumulation in one or more organs of a human subject having an acid sphingomyelinase deficiency (ASMD), comprising: (a) administering at least one initial dose of recombinant human acid sphingomyelinase (rhASM) to the human subject, wherein the initial dose is 0.3 mg/kg; and (b) subsequent to the administration of the at least one initial dose, administering a higher dose of rhASM to the human subject if the human subject has a total bilirubin concentration of less than or equal to 2.1 mg/dL and the human subject does not experience a severe related adverse event, wherein the higher dose is from 0.1 mg/kg to 3.0 mg/kg higher than the initial dose.