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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Detalles Bibliográficos
Autores principales: Dornelles, Alícia Dorneles, Junges, Ana Paula Pedroso, Pereira, Tiago Veiga, Krug, Bárbara Corrêa, Gonçalves, Candice Beatriz Treter, Llerena, Juan Clinton, Kishnani, Priya Sunil, de Oliveira, Haliton Alves, Schwartz, Ida Vanessa Doederlein
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2021
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8584814/
https://www.ncbi.nlm.nih.gov/pubmed/34768348
http://dx.doi.org/10.3390/jcm10214828

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8584814/
https://www.ncbi.nlm.nih.gov/pubmed/34768348
http://dx.doi.org/10.3390/jcm10214828

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