Gaucher disease (GD), the most prevalent lysosomal storage disease, is caused by a
deficiency of glucocerebrosidase (GCase). The identification of small molecules acting as
agents for enzyme enhancement therapy is an attractive approach for treating different forms
of GD. A thermal denaturation assay utilizing wild type GCase was developed to screen a
library of 1,040 Food and Drug Administration-approved drugs. Ambroxol (ABX), a drug
used to treat airway mucus hypersecretion and hyaline membrane disease in newborns …