Investigators obtained informed, written consent from each patient and/or the parent or guardian. Patients were randomized to receive taliglucerase alfa 30 U/kg selleckchem or 60 U/kg per infusion every other week for 12 months.

The primary end point was the median percent change in hemoglobin concentration from baseline and the interquartile range of median percent change in hemoglobin levels from baseline. Secondary end points included the percent changes from baseline in spleen volume, liver volume, platelet counts, and either chitotriosidase or CCL18 activity. Exploratory end points of organ volumes expressed as multiples of normal (MN) were calculated using normal spleen volume = 2 mL/kg multiplied by body weight in kg and normal liver volume = 25 mL/kg multiplied by body weight in kg. Exploratory end points included: change in height, weight, puberty, and bone age (based on radiograph of the left hand and wrist); occurrence of bone events including bone crises; and quality of life using Child Health Questionnaire™ (CHQ) PF-28 (valid for patients aged 5 to 18 years). Safety end points included AEs and changes in clinical laboratory

findings, echocardiographic readings, and anti-taliglucerase alfa antibody titers. Occurrence of bone events, including bone crises, was part of the analysis of AEs. Male and female patients aged 2 to < 18 years were required to have a diagnosis of GD with leukocyte acid beta-glucocerebrosidase activity level ≤ 30% of the mean activity of the reference range for healthy individuals. Patients were eligible if they had not Vemurafenib in vivo received ERT in the past or within the previous 12 months and had a negative anti-glucocerebrosidase assay assessment, had not received substrate reduction therapy for GD in the past 12 months, and were judged in need of treatment with ERT based on clinical condition and the opinion of the local investigator. Gefitinib Patients were excluded based on any of the following criteria: presence of complex neuronopathic features other than longstanding

oculomotor gaze palsy; unresolved anemia due to iron, folic acid, or vitamin B12 deficiency; currently taking another investigational drug for any condition; previous hypersensitive reaction to alglucerase or imiglucerase; history of allergy to carrots; inability of parents or guardians to understand the nature, scope, and consequences of study participation; and presence of any medical, behavioral, psychological/emotional condition that would, in the investigator’s opinion, interfere with full participation in the study. Spleen and liver volumes were measured using magnetic resonance imaging as previously reported [15] and were assessed at BioClinica, Lyon, France. Beta-glucocerebrosidase activity, chitotriosidase or CCL18 activity, and DNA sequencing were performed at a centralized laboratory, the Academic Medical Center in Amsterdam, The Netherlands.