GBA1 and lysosomal storage disease: The hypothesis that ambroxol (ABX) could potentially be useful in DLB originated in studies on Gaucher disease (GD), a lysosomal storage disorder, caused by a mutation in the glucocerebrosidase (GCase) gene (GBA1) and resulting in the development of parkinsonism in some patients (Tayebi et al., 2003).