A Systematic Review and Meta-Analysis of Enzyme Replacement Therapy in Late-Onset Pompe Disease

A Systematic Review and Meta-Analysis of Enzyme Replacement Therapy in Late-Onset Pompe Disease

Pompe disease (PD) is a glycogen storage disorder caused by 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 late-onset PD (LOPD). Methods: We systematically...

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Autores principales: Alícia Dorneles Dornelles, Ana Paula Pedroso Junges, Tiago Veiga Pereira, Bárbara Corrêa Krug, Candice Beatriz Treter Gonçalves, Juan Clinton Llerena, Priya Sunil Kishnani, Haliton Alves de Oliveira, Ida Vanessa Doederlein Schwartz
Formato: article
Lenguaje:EN
Publicado: MDPI AG 2021
Materias:
glycogen storage disease type II
alpha-glucosidase
Pompe disease
enzyme replacement therapy
Medicine
R
Acceso en línea:https://doaj.org/article/1c5d900c95db4428981b0e9388357da8
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https://doaj.org/article/1c5d900c95db4428981b0e9388357da8

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