Effectiveness of enzyme replacement therapy in adults with late-onset Pompe disease: results from the NCS-LSD cohort study.
Anderson, L J
Wyatt, K M
Hughes, D A
Lachmann, R H
JournalJournal of inherited metabolic disease
Research Support, Non-U.S. Gov't
RightsArchived with thanks to Journal of inherited metabolic disease
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To determine the effectiveness of enzyme replacement therapy (ERT) for adults with late-onset Pompe disease.