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Common presenting signs include thrombocytopenia, anemia, hepatosplenomegaly, bone abnormalities, and, additionally in children, growth failure. Fifty\u2010seven patients aged 3\u201362 years at the baseline of two phase III trials for velaglucerase alfa treatment were enrolled in the single extension study. In the extension, they received every\u2010other\u2010week velaglucerase alfa intravenous infusions for 1.2\u20134.8 years at 60 U\/kg, although 10 patients experienced dose reduction. No patient experienced a drug\u2010related serious adverse event or withdrew due to an adverse event. One patient died following a convulsion that was reported as unrelated to the study drug. Only one patient tested positive for anti\u2010velaglucerase alfa antibodies. Combining the experience of the initial phase III trials and the extension study, significant improvements were observed in the first 24 months from baseline in hematology variables, organ volumes, plasma biomarkers, and, in adults, the lumbar spine bone mineral density Z\u2010score. Improvements were maintained over longer\u2010term treatment. Velaglucerase alfa had a good long\u2010term safety and tolerability profile, and patients continued to respond clinically, which is consistent with the results of the extension study to the phase I\/II trial of velaglucerase alfa. EudraCT number 2008\u2010001965\u201027;\n                    <jats:ext-link xmlns:xlink=\"http:\/\/www.w3.org\/1999\/xlink\" xlink:href=\"http:\/\/www.clinicaltrials.gov\">www.clinicaltrials.gov<\/jats:ext-link>\n                    identifier NCT00635427. Am. J. 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