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Title: Enzyme replacement therapy in pediatric patients with Gaucher disease: what should we use as maintenance dosage? Author: Brunel-Guitton C, Rivard GE, Galipeau J, Alos N, Miron MC, Therrien R, Mitchell G, Lapierre G, Lambert M. Journal: Mol Genet Metab; 2009 Feb; 96(2):73-6. PubMed ID: 19083253. Abstract: INTRODUCTION: No consensus exists on the minimal dose of enzyme replacement therapy (ERT) effective to maintain therapeutic goals in pediatric Gaucher patients. OBJECTIVE: Evaluate the efficacy of low dosage ERT to maintain treatment goals. RESULTS: Six patients had a maintenance dose of 30-35U/kg/month. All patients, with the exception of one L444P/L444P homozygote, maintained therapeutic goals. DISCUSSION: A low maintenance dose may be adequate in most pediatric patients. L444P homozygotes may require a higher maintenance dosage.[Abstract] [Full Text] [Related] [New Search]