Abstract
Enzyme Replacement Therapy (ERT) for Pompe disease was approved for clinical use in 2006 based on the efficacy of alglucosidase alfa in classic infantile patients. More recently, treatment efficacy was demonstrated in adults. We evaluated the long-term effects of ERT and compared effects with the natural disease course. Furthermore prognostic factors for treatment response were identified. In this ongoing prospective study, we included 71 adults who were treated with 20mg/kg alglucosidase alfa every other week.
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