Efficacy and safety of enzyme replacement therapy with alglucosidase alfa for the treatment of patients with infantile-onset Pompe disease: a systematic review and metanalysis

Efficacy and safety of enzyme replacement therapy with alglucosidase alfa for the treatment of patients with infantile-onset Pompe disease: a systematic review and metanalysis

IntroductionPompe disease (PD) is a glycogen disorder caused by the deficient activity of acid alpha-glucosidase (GAA). We sought to review the latest available evidence on the safety and efficacy of recombinant human GAA enzyme replacement therapy (ERT) for infantile-onset PD (IOPD).MethodsWe syste...

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Bibliographic Details
Main Authors:	A. D. Dornelles, A. P. P. Junges, B. Krug, C. Gonçalves, H. A. de Oliveira Junior, I. V. D. Schwartz
Format:	Article
Language:	English
Published:	Frontiers Media S.A. 2024-02-01
Series:	Frontiers in Pediatrics
Subjects:	enzyme replacement therapy alglucosidase alfa Pompe disease glycogen storage disease type II systematic review meta-analysis
Online Access:	https://www.frontiersin.org/articles/10.3389/fped.2024.1310317/full

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