DOSE ESCALATION ENZYME REPLACEMENT THERAPY FOR TREATING ACID SPHINGOMYELINASE DEFICIENCY

The invention relates to a recombinant human acid sphingomyelinase (rhASM) for use in a method of treating an acid sphingomyelinase deficiency (ASMD) in a human subject in need thereof, the method comprising administering to the human subject at least one initial dose of 0.025 mg/kg to 0.05 mg/kg of said rhASM; and administering to the human subject an escalating dose regimen at the following sequential doses: (i) 0.1 mg/kg of said rhASM, (ii) 0.3 mg/kg of said rhASM, and (iii) 0.6 mg/kg of said rhASM, wherein each escalating dose is administered at least once before elevating the dose to the next level.