Muscle imaging data in late-onset Pompe disease reveal a correlation between the pre-existing degree of lipomatous muscle alterations and the efficacy of long-term enzyme replacement therapy

BACKGROUND: Late-onset Pompe disease (LOPD) is a metabolic myopathy caused by mutations in GAA and characterized by proximal muscle weakness and respiratory insufficiency. There is evidence from clinical studies that enzyme replacement therapy (ERT) with human recombinant alpha-glucosidase improves...

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Detalles Bibliográficos
Autores principales: Gruhn, Kai Michael, Heyer, Christoph Malte, Güttsches, Anne-Katrin, Rehmann, Robert, Nicolas, Volkmar, Schmidt-Wilcke, Tobias, Tegenthoff, Martin, Vorgerd, Matthias, Kley, Rudolf Andre
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2015
Materias:
SI:Therapy
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4750633/
https://www.ncbi.nlm.nih.gov/pubmed/26937398
http://dx.doi.org/10.1016/j.ymgmr.2015.03.010

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