Clinical efficacy of the enzyme replacement therapy in patients with late-onset Pompe disease: a systematic review and a meta-analysis

In patients with late-onset Pompe disease (LOPD), the efficacy of the enzyme replacement therapy (ERT) with recombinant human alpha-glucosidase (rhGAA) is difficult to evaluate, due to the clinical heterogeneity and the small sample sizes in published studies. Therefore, we conduct a systematic lite...

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Detalles Bibliográficos
Autores principales: Sarah, Berli, Giovanna, Brandi, Emanuela, Keller, Nadi, Najia, Josè, Vitale, Alberto, Pagnamenta
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer Berlin Heidelberg 2021
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8782782/
https://www.ncbi.nlm.nih.gov/pubmed/33851281
http://dx.doi.org/10.1007/s00415-021-10526-5

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8782782/
https://www.ncbi.nlm.nih.gov/pubmed/33851281
http://dx.doi.org/10.1007/s00415-021-10526-5

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