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Home-Based Infusion of Alglucosidase Alfa Can Safely be Implemented in Adults with Late-Onset Pompe Disease: Lessons Learned from 18,380 Infusions

Ditters, IAM; van Kooten, HA; van der Beek, NAME; Hardon, JF; Ismailova, G; Brusse, E; Kruijshaar, ME; van der Ploeg, AT; van den Hout, JMP; Huidekoper, HH

van der Ploeg, AT (通讯作者),Erasmus MC, Univ Med Ctr, Ctr Lysosomal & Metab Dis, Dept Pediat,Sophia Childrens Hosp, OB 2060, NL-3000 CB Rotterdam, Netherlands.

BIODRUGS, 2023; 37 (5): 685

Abstract

BackgroundEnzyme replacement therapy (ERT) with alglucosidase alfa is the treatment for patients with Pompe disease, a hereditary metabolic myopathy. ......

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