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Article

Effect of enzyme therapy and prognostic factors in 69 adults with Pompe disease: an open-label single-center study

26 Sept 2012

Abstract excerpt

BACKGROUND: Enzyme replacement therapy (ERT) in adults with Pompe disease, a progressive neuromuscular disorder, is of promising but variable efficacy. We investigated whether it alters the course of disease, and also identified potential prognostic factors. METHODS: Patients in this open-label single-center study were treated biweekly with 20 mg/kg alglucosidase alfa. Muscle strength, muscle function, and...

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PMID: 23013746
DOI: 10.1186/1750-1172-7-73

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