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Erschienen in: Journal of Inherited Metabolic Disease 5/2009

01.10.2009 | Original Article

Management of neuronopathic Gaucher disease: Revised recommendations

verfasst von: A. Vellodi, A. Tylki-Szymanska, E. H. Davies, E. Kolodny, B. Bembi, T. Collin-Histed, E. Mengel, A. Erikson, R. Schiffmann

Erschienen in: Journal of Inherited Metabolic Disease | Ausgabe 5/2009

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Summary

The original guidelines drawn up for the management of the neuronopathic forms of Gaucher disease were felt to be in need of revision; in particular, the role of high-dose enzyme replacement therapy (120 IU/kg of body weight every 2 weeks) in stabilizing neurological disease. The existing published evidence was analysed; it was concluded that it did not support the role of high-dose ERT, although this might be required to treat severe visceral disease.
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Metadaten
Titel
Management of neuronopathic Gaucher disease: Revised recommendations
verfasst von
A. Vellodi
A. Tylki-Szymanska
E. H. Davies
E. Kolodny
B. Bembi
T. Collin-Histed
E. Mengel
A. Erikson
R. Schiffmann
Publikationsdatum
01.10.2009
Verlag
Springer Netherlands
Erschienen in
Journal of Inherited Metabolic Disease / Ausgabe 5/2009
Print ISSN: 0141-8955
Elektronische ISSN: 1573-2665
DOI
https://doi.org/10.1007/s10545-009-1164-2

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