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

Internet

https://www.frontiersin.org/articles/10.3389/fped.2024.1310317/full

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