Dose escalation enzyme replacement therapy for treating acid sphingomyelinase deficiency
AssigneesMount Sinai School of Medicine, 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.
Claims26
A method for treating a human subject having an acid sphingomyelinase deficiency, comprising administering recombinant human acid sphingomyelinase (rhASM) in an escalating dose regimen at the following sequential doses:
- (a) 0.1 mg/kg,
- (b) 0.3 mg/kg, and
- (c) 0.6 mg/kg;
- wherein each dose of rhASM is administered at two week intervals, wherein each dose is administered at least once before elevating the dose to the next level, and wherein the subject is monitored for toxic side effects before elevating the dose to the next level.
Dependencies are inferred from the claim text.