Dataset from A Multicenter, Double-blind, Randomized Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease

This is a multi-center, double-blind trial to assess the safety and efficacy of taliglucerase
alfa in untreated subjects (2 to <18 years old) with Gaucher disease randomly assigned to
treatment with one of two doses, 30 or 60 units/kg. Subjects will receive an intravenous (IV)
infusion of taliglucerase alfa every two weeks. The total duration of treatment will be 12
months. At the end of the 12-month treatment period eligible subjects will be offered
enrollment in an open-label extension study if taliglucerase alfa is not commercially
available.