Severe distal muscle involvement and mild sensory neuropathy in a boy with infantile onset Pompe disease treated with enzyme replacement therapy for 6 years.

Anne Schänzer,Jonas Görlach, Kerstin Claudi,Andreas Hahn

Neuromuscular Disorders（2019）

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摘要

•Weakness of the feet dorsiflexors can evolve in IOPD after long-term ERT.•This distal weakness is due to progression of the myopathy.•A neuropathy in IOPD constitutes a further symptom that may evolve despite ERT.

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关键词

IOPD,ERT,Infantile-onset Pompe disease,Pompe disease,GAA,IENFD

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