Long-term effectiveness of enzyme replacement therapy in children with Gaucher disease: results from the NCS-LSD cohort study.
Author
Anderson, L. J.
Henley, W. E.
Wyatt, K. M.
Nikolaou, V.
Waldek, S.
Hughes, D. A.
Pastores, G. M.
Logan, Stuart
Date
2014-11Journal
Journal of inherited metabolic diseaseType
Journal ArticleMulticenter Study
Observational Study
Research Support, Non-U.S. Gov't
Publisher
SpringerDOI
10.1007/s10545-014-9693-8Rights
Archived with thanks to Journal of inherited metabolic diseaseMetadata
Show full item recordAbstract
To determine the effectiveness of enzyme replacement therapies (ERT) for children with Gaucher disease (GD).