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035, 95% CI 1.0, 25. from baseline to last observation. When stratified by pediatric versus adult patients, there was improvement in BOT-2 total point score in patients less then 18 years (mean percent increase from baseline to last observation 23%) compared to adults (mean decrease of -0.7%). Subtest analysis of individual BOT-2 items captured some improvement following velmanase alfa treatment in pediatric patients. Conclusions There was limited ability to assess the BOT-2 change responses in adults. Pediatric patients showed stabili
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