A multicenter, open-label, phase III study of Abcertin in Gaucher disease

AbstractBackground:Gaucher disease (GD) is caused by a deficiency in the lysosomal enzyme glucocerebrosidase. Enzyme replacement therapy (ERT) is recommended for clinical improvement.Methods:The efficacy and safety of a new imiglucerase, Abcertin, were assessed in 7 Egyptian patients with treatment-