Acid sphingomyelinase deficiency (ASMD) is a rare genetic disorder caused by mutations in the sphingomyelin phosphodiesterase 1 (SMPD1) gene. The mutations cause a deficiency of acid sphingomyelinase enzyme (ASM), which normally breaks down sphingomyelin to ceramide and phosphocholine.
As a result, sphingomyelin accumulates in lysosomes in the cells of the liver, lung, spleen, and brain. Olipudase alfa An enzyme replacement therapy that acts as an ASM Available as lyophilized powder and a single-dose vial Intravenous administration Dosage and administration Before initiation of olipudase alfa Evaluate baseline levels of alanine aminotransferase (ALT) and aspartate aminotransferase (AST) within one month before initiating the treatment Verify pregnancy status in female patients Pretreat patients with antipyretics, antihistamines, and/or corticosteroids before administering olipudase alfa…
