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

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URI
http://hdl.handle.net/11287/617986
Author
Anderson, L. J.
Henley, W. E.
Wyatt, K. M.
Nikolaou, V.
Waldek, S.
Hughes, D. A.
Pastores, G. M.
Logan, Stuart
Date
2014-11
Journal
Journal of inherited metabolic disease
Type
Journal Article
Multicenter Study
Observational Study
Research Support, Non-U.S. Gov't
Publisher
Springer
DOI
10.1007/s10545-014-9693-8
Rights
Archived with thanks to Journal of inherited metabolic disease
Metadata
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Abstract
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.
Publisher URL
http://dx.doi.org/10.1007/s10545-014-9693-8
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