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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Detalles Bibliográficos
Autores principales: A. D. Dornelles, A. P. P. Junges, B. Krug, C. Gonçalves, H. A. de Oliveira Junior, I. V. D. Schwartz
Formato: Artículo
Lenguaje:English
Publicado: Frontiers Media S.A. 2024-02-01
Colección:Frontiers in Pediatrics
Materias:
enzyme replacement therapy
alglucosidase alfa
Pompe disease
glycogen storage disease type II
systematic review
meta-analysis
Acceso en línea:https://www.frontiersin.org/articles/10.3389/fped.2024.1310317/full

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