[HTML][HTML] Novel first-dose adverse drug reactions during a phase I trial of olipudase alfa (recombinant human acid sphingomyelinase) in adults with Niemann–Pick …
MM McGovern, MP Wasserstein, B Kirmse… - Genetics in …, 2016 - Elsevier
Purpose Enzyme replacement therapy with olipudase alfa (recombinant human acid
sphingomyelinase) is being developed for Niemann–Pick disease type B (NPD B). Methods
A single-center, open-label, nonrandomized, single-ascending-dose trial evaluated the
safety of intravenous olipudase alfa (0.03–1.0 mg/kg) in 11 adults with NPD B. Patients were
monitored in the hospital for 72 h after infusion and had follow-up visits on days 14 and 28.
Results Plasma ceramide, a product of sphingomyelin catabolism by olipudase alfa, showed …
sphingomyelinase) is being developed for Niemann–Pick disease type B (NPD B). Methods
A single-center, open-label, nonrandomized, single-ascending-dose trial evaluated the
safety of intravenous olipudase alfa (0.03–1.0 mg/kg) in 11 adults with NPD B. Patients were
monitored in the hospital for 72 h after infusion and had follow-up visits on days 14 and 28.
Results Plasma ceramide, a product of sphingomyelin catabolism by olipudase alfa, showed …