GBA1 and Gaucher disease type 1: Disappointing results were also seen in a phase 2 trial with the pharmacological chaperone Plicera (afegostat tartrate) in the treatment of type 1 Gaucher disease: whereas all patients enrolled in this study experienced an increase in the level of the target enzyme glucocerebrosidase as measured in white blood cells, clinically meaningful improvement was recorded in just one out of the eighteen patients completing who completed the study [19].