[HTML][HTML] Nonclinical safety assessment of recombinant human acid sphingomyelinase (rhASM) for the treatment of acid sphingomyelinase deficiency: the utility of …
JM Murray, AM Thompson, A Vitsky, M Hawes… - Molecular Genetics and …, 2015 - Elsevier
Recombinant human acid sphingomyelinase (rhASM) is being developed as an enzyme
replacement therapy for patients with acid sphingomyelinase deficiency (Niemann–Pick
disease types A and B), which causes sphingomyelin to accumulate in lysosomes. In the
acid sphingomyelinase knock-out (ASMKO) mouse, intravenously administered rhASM
reduced tissue sphingomyelin levels in a dose-dependent manner. When rhASM was
administered to normal rats, mice, and dogs, no toxicity was observed up to a dose of 30 …
replacement therapy for patients with acid sphingomyelinase deficiency (Niemann–Pick
disease types A and B), which causes sphingomyelin to accumulate in lysosomes. In the
acid sphingomyelinase knock-out (ASMKO) mouse, intravenously administered rhASM
reduced tissue sphingomyelin levels in a dose-dependent manner. When rhASM was
administered to normal rats, mice, and dogs, no toxicity was observed up to a dose of 30 …
