Review of miglustat for clinical management in Gaucher disease type 1
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چکیده
منابع مشابه
Review of miglustat for clinical management in Gaucher disease type 1
Gaucher disease is a progressive lysosomal storage disorder caused by the deficiency of glucocerebrosidase, and characterized by intralysosomal storage of glucosylceramide that leads to dysfunction in multiple organ systems. Intravenous enzyme replacement with imiglucerase is the accepted standard for treatment of symptomatic patients and has been effective in reducing many of the signs and sym...
متن کاملclinical manifestations of type 1 gaucher disease
how to cite this article: salehpour sh. clinical manifestations of type 1 gaucher disease. iran j child neurol autumn 2012; 6:4 (suppl. 1):13-14. pls see pdf. refe r ences 1. beutler e, grabowski ga. gaucher disease. in: metabolic and molecular bases of inherited disease, scriver cr, beaudet al, sly ws, valle d (eds), mcgraw-hill, new york 2001: 3635. 2. cox tm, schofield jp. gaucher’s diseas...
متن کاملChanges in the atherogenic profile of patients with type 1 Gaucher disease after miglustat therapy.
OBJECTIVE Type 1 Gaucher disease (GD1) is an autosomal recessive lysosomal storage disorder associated with abnormal accumulation of glucocerebrosides. Plasma total cholesterol (TC), low-density lipoprotein cholesterol (LDL-c), and high-density lipoprotein cholesterol (HDL-c) are decreased in GD1 patients. The effects of substrate reduction therapy (SRT) with miglustat on plasma lipids and athe...
متن کاملReal-world clinical experience with long-term miglustat maintenance therapy in type 1 Gaucher disease: the ZAGAL project.
There are few published data from real-world clinical experience with miglustat (Zavesca), an oral inhibitor of glucosylceramide synthase, in type 1 Gaucher disease. We report data from a prospective, open-label investigational study that evaluated substrate reduction therapy with miglustat 100 mg t.i.d. as a maintenance therapy in patients with Type 1 Gaucher disease who had been switched from...
متن کاملGoal-oriented therapy with miglustat in Gaucher disease.
BACKGROUND Gaucher disease (GD) is a highly heterogeneous disorder with multisystem involvement. Specific therapeutic goals for each manifestation of type 1 GD (GD1) were established in 2004 by an international panel of experts, to facilitate better management of GD1 patients. The goals were defined based on experience with enzyme replacement therapy (ERT) using imiglucerase. Miglustat, a small...
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ژورنال
عنوان ژورنال: Therapeutics and Clinical Risk Management
سال: 2008
ISSN: 1178-203X
DOI: 10.2147/tcrm.s6865