Twice weekly dosing with Sebelipase alfa (Kanuma®) rescues severely ill infants with Wolman disease.
María José de CastroSimon A JonesJavier de Las HerasPaula Sánchez-PintosMaría L CouceCristóbal ColónPablo CrujeirasMaría UncetaHeather ChurchKathryn BrammeierWu Hoi YeeJames CooperLaura López de FrutosIrene Serrano-GonzaloMaría José CambaFiona J WhiteVictoria HolmesArunabha GhoshPublished in: Orphanet journal of rare diseases (2024)
5 mg/kg twice weekly dosing with Sebelipase alfa rescues severely ill infants with WD by increasing substrate clearance. There is biologically relevant lipid accumulation in the 'trough' periods before the next dosing, even with this intensive regimen.
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