Long-term follow-up of 17 patients with childhood Pompe disease treated with enzyme replacement therapy

OBJECTIVES: Pompe disease is a progressive metabolic myopathy for which enzyme replacement therapy (ERT) was approved in 2006. While various publications have examined the effects of ERT in classic-infantile patients and in adults, little has been published on ERT in children with non-classic presen...

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Detalles Bibliográficos
Autores principales: van der Meijden, Jan C., Kruijshaar, Michelle E., Harlaar, Laurike, Rizopoulos, Dimitris, van der Beek, Nadine A. M. E., van der Ploeg, Ans T.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer Netherlands 2018
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6326992/
https://www.ncbi.nlm.nih.gov/pubmed/29556838
http://dx.doi.org/10.1007/s10545-018-0166-3

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