[HTML][HTML] Successful within-patient dose escalation of olipudase alfa in acid sphingomyelinase deficiency
Background Olipudase alfa, a recombinant human acid sphingomyelinase (rhASM), is an
investigational enzyme replacement therapy (ERT) for patients with ASM deficiency [ASMD;
Niemann–Pick Disease (NPD) A and B]. This open-label phase 1b study assessed the
safety and tolerability of olipudase alfa using within-patient dose escalation to gradually
debulk accumulated sphingomyelin and mitigate the rapid production of metabolites, which
can be toxic. Secondary objectives were pharmacokinetics, pharmacodynamics, and …
investigational enzyme replacement therapy (ERT) for patients with ASM deficiency [ASMD;
Niemann–Pick Disease (NPD) A and B]. This open-label phase 1b study assessed the
safety and tolerability of olipudase alfa using within-patient dose escalation to gradually
debulk accumulated sphingomyelin and mitigate the rapid production of metabolites, which
can be toxic. Secondary objectives were pharmacokinetics, pharmacodynamics, and …