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Convection perfusion of glucocerebrosidase for neuronopathic Gaucher's disease

✍ Scribed by Russell R. Lonser; Stuart Walbridge; Gary J. Murray; Michele R. Aizenberg; Alexander O. Vortmeyer; Johannes M. F. G. Aerts; Roscoe O. Brady; Edward H. Oldfield


Publisher
John Wiley and Sons
Year
2005
Tongue
English
Weight
213 KB
Volume
57
Category
Article
ISSN
0364-5134

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We prospectively evaluated the clinical and biochemical responses to enzyme-replacement therapy (ERT) with macrophage-targeted glucocerebrosidase (Ceredase) infusions in 5 patients (age, 3.5-8.5 years) with type 3 Gaucher's disease. The patients were followed for up to 5 years. Enzyme dosage ranged