Erschienen in:
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.