2019Neuromuscular DisordersRequires access

P.71The emerging phenotype in long-term enzyme-treated classic infantile Pompe patients: severe distal muscle weakness

J. van den Dorpel, Esther Poelman, Laurike Harlaar, H. van Kooten, Lianne van der Giessen, P. van Doorn, A. van der Ploeg, J. van den Hout, N. van der Beek

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Key concepts: Weakness, Medicine, Proximal muscle weakness, Muscle weakness, Myopathy, Congenital myopathy, Glycogen storage disease type II, Enzyme replacement therapy

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P.71The emerging phenotype in long-term enzyme-treated classic infantile Pompe patients: severe distal muscle weakness — Research Paper | ScholarLens