Improvement of dysphagia in a child affected by Pompe disease treated with enzyme replacement therapy

AIM: Dysphagia is a known complication in Pompe Disease (PD), a severe metabolic myopathy due to alpha-glucosidase deficiency. Enzyme replacement therapy (ERT) with alglucosidase alfa is the only approved therapy for PD. Presently no data are available on the effects of ERT on dysphagia in PD patien...

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Detalhes bibliográficos
Main Authors: Fecarotta, Simona, Ascione, Serena, Montefusco, Giuseppe, Della Casa, Roberto, Villari, Paola, Romano, Alfonso, Del Giudice, Ennio, Andria, Generoso, Parenti, Giancarlo
Formato: Artigo
Idioma:Inglês
Publicado em: BioMed Central 2013
Assuntos:
Case Report
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3667015/
https://ncbi.nlm.nih.gov/pubmed/23668440
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/1824-7288-39-30
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