Dose escalation enzyme replacement therapy for treating acid sphingomyelinase deficiency
AssigneesIcahn School of Medicine at Mount Sinai, Genzyme Corporation
InventorsEdward H. Schuchman, Robert J. Desnick, Gerald F. Cox, Laura P. Andrews, James M. Murray
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Abstract
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.
Claims13
A method for treating 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 from 0.025 mg/kg to 0.275 mg/kg;
- and
- (b) if the plasma ceramide concentration of the human subject is equal to or less than 10 μg/mL after the administration of the at least one initial dose, administering a higher dose of rhASM to the human subject.
A method for treating 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 from 0.025 mg/kg to 0.275 mg/kg;
- and
- (b) if the total bilirubin concentration of the human subject is equal to or less than 1.5 mg/dL after the administration of the at least one initial dose, administering a higher dose of rhASM to the human subject.
A method for treating 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 from 0.025 mg/kg to 0.275 mg/kg;
- and
- (b) if the plasma C-reactive protein (CRP) concentration of the human subject is equal to or less than 12 mg/L after the administration of the at least one initial dose, administering a higher dose of rhASM to the human subject.
Dependencies are inferred from the claim text.