Taliglucerase alfa is a plant cell-expressed beta-glucocerebrosidase approved in 
the United States, Israel, Australia, Canada, and other countries for enzyme 
replacement therapy in adults with Type 1 Gaucher disease (GD), for treatment of 
pediatric patients in the United States, Australia, and Canada, and for the 
hematologic manifestations of Type 3 GD in pediatric patients in Canada. This 
multicenter, randomized, double-blind, parallel-dose, 12-month study assessed 
efficacy and safety of taliglucerase alfa in pediatric patients with GD. Eleven 
children were randomized to taliglucerase alfa 30U/kg (n=6) or 60U/kg (n=5) per 
infusion every other week. From baseline to month 12, the following changes were 
noted in the taliglucerase alfa 30-U/kg and 60-U/kg dose groups, respectively: 
median hemoglobin concentrations increased by 12.2% and 14.2%; the interquartile 
ranges of median percent change in hemoglobin levels from baseline were 20.6 and 
10.4, respectively; mean spleen volume decreased from 22.2 to 14.0 multiples of 
normal (MN) and from 29.4 to 12.9 MN; mean liver volume decreased from 1.8 to 
1.5 MN and from 2.2 to 1.7 MN; platelet counts increased by 30.9% and 73.7%; and 
chitotriosidase activity was reduced by 58.5% and 66.1%. Nearly all adverse 
events were mild/moderate, unrelated to treatment, and transient. One patient 
presented with treatment-related gastroenteritis reported as a serious adverse 
event due to the need for hospitalization for rehydration. No patient 
discontinued. These data suggest that taliglucerase alfa has the potential to be 
a therapeutic treatment option for children with GD. This study was registered 
at www.clinicaltrials.gov as NCT01132690.