Twice weekly dosing with Sebelipase alfa (Kanuma®) rescues severely ill infants with Wolman disease
María José de Castro,
Simon A Jones,
Javier de las Heras
et al.
Abstract:Background
Sebelipase alfa (Kanuma®) is approved for patients with Wolman disease (WD) at a dosage of 3–5 mg/kg once weekly. Survival rates in the second of two clinical trials was greater, despite recruiting more severely ill patients, probably related to higher initial and maximal doses. We aimed to evaluate the effective pharmacokinetics and pharmacodynamics of Sebelipase alfa when administered to patients with severe WD at 5 mg/kg twice weekly, an intensive regimen which was not assessed in… Show more
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