Long-term effectiveness of enzyme replacement therapy in children with Gaucher disease: results from the NCS-LSD cohort study.
Anderson, L. J.
Henley, W. E.
Wyatt, K. M.
Hughes, D. A.
Pastores, G. M.
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 therapies (ERT) for children with Gaucher disease (GD).