Abstract
Enzyme replacement therapy (ERT) has substantially improved the prognosis in classic-infantile Pompe patients. Different dosing regimens have been reported in the literature, but no study comparing the effects of different dosing regimens in a larger cohort of classic-infantile Pompe patients has been published. The aim of this study was to obtain an overview of dosing regimens and treatment outcome within the European Pompe Consortium (EPOC). A retrospective, multicentre study was conducted, including 116 confirmed classic-infantile patients treated with ERT from France, Germany, Italy and the Netherlands.
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