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.
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| http://dx.doi.org/10.1016/j.ymgme.2008.11.158 | DOI Listing |
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