Taliglucerase alfa is an enzyme replacement therapy approved for treatment of 
Gaucher disease (GD) in children and adults in several countries. This 
multicenter extension study assessed the efficacy and safety of taliglucerase 
alfa in pediatric patients with GD who were treatment-naïve (n=10) or switched 
from imiglucerase (n=5). Patients received taliglucerase alfa 30 or 60U/kg 
(treatment-naïve) or the same dose as previously treated with imiglucerase every 
other week. In treatment-naïve patients, taliglucerase alfa 30 and 60U/kg, 
respectively, reduced mean spleen volume (-18.6 multiples of normal [MN] and 
-26.0MN), liver volume (-0.8MN and -0.9MN), and chitotriosidase activity (-72.7% 
and -84.4%), and increased mean Hb concentration (+2.0g/dL and +2.3g/dL) and 
mean platelet count (+38,200/mm3 and +138,250/mm3) from baseline through 36 
total months of treatment. In patients previously treated with imiglucerase, 
these disease parameters remained stable through 33 total months of treatment 
with taliglucerase alfa. Most adverse events were mild/moderate; treatment was 
well tolerated. These findings extend the taliglucerase alfa safety and efficacy 
profile and demonstrate long-term clinical improvement in treatment-naïve 
children receiving taliglucerase alfa and maintenance of disease stability in 
children switched to taliglucerase alfa. Treatment was well-tolerated, with no 
new safety signals. This study is registered at www.clinicaltrials.gov as 
NCT01411228.