Distal muscle weakness is a common and early feature in long-term enzyme-treated classic infantile Pompe patients

BACKGROUND: Enzyme replacement therapy (ERT; alglucosidase alfa) has improved the prospects for patients with classic infantile Pompe disease considerably. However, over time we noticed that many of these children exhibit distal muscle weakness at an early age, which is in contrast to the primarily...

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Detalles Bibliográficos
Autores principales: van den Dorpel, J. J. A., Poelman, E., Harlaar, L., van Kooten, H. A., van der Giessen, L. J., van Doorn, P. A., van der Ploeg, A. T., van den Hout, J. M. P., van der Beek, N. A. M. E.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2020
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7488760/
https://www.ncbi.nlm.nih.gov/pubmed/32928284
http://dx.doi.org/10.1186/s13023-020-01482-w

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