Management of neuronopathic Gaucher disease: revised recommendations
2009 (English)In: Journal of Inherited Metabolic Disease, ISSN 0141-8955, E-ISSN 1573-2665, Vol. 32, no 5, 660-664 p.Article in journal (Refereed) Published
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.
Place, publisher, year, edition, pages
Springer, 2009. Vol. 32, no 5, 660-664 p.
neuronopathic, Gaucher disease, revised recommendations
IdentifiersURN: urn:nbn:se:umu:diva-35056DOI: 10.1007/s10545-009-1164-2PubMedID: 19655269OAI: oai:DiVA.org:umu-35056DiVA: diva2:328508