Long-term effectiveness of enzyme replacement therapy in children with Gaucher disease: results from the NCS-LSD cohort study.

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Authors
Anderson, L. J.
Henley, W. E.
Wyatt, K. M.
Nikolaou, V.
Waldek, S.
Hughes, D. A.
Pastores, G. M.
Logan, Stuart
Journal
Journal of inherited metabolic disease
Type
Journal Article
Multicenter Study
Observational Study
Research Support, Non-U.S. Gov't
Publisher
Springer
Rights
Archived with thanks to Journal of inherited metabolic disease
To determine the effectiveness of enzyme replacement therapies (ERT) for children with Gaucher disease (GD).
Citation
Long-term effectiveness of enzyme replacement therapy in children with Gaucher disease: results from the NCS-LSD cohort study. 2014, 37 (6):961-8 J. Inherit. Metab. Dis.
Note