ML266 is a glucocerebrosidase activator and a non-inhibitory chaperone of glucocerebrosidase.

LTI-291, a extract from patent WO 2017192841 A1, is an activator of glucocerebrosidase (Gcase) for treatment of Parkinson's disease.

Afegostat, aslo known as Isofagomine, and AT2101, was an experimental drug for the treatment of certain forms of Gaucher's disease. Isofagomine (IFG) is an acid β-glucosidase (GCase) active site inhibitor that acts as a pharmacological chaperone. FG inhibits GCase with K(i) ~30 nM for wild-type and mutant enzymes (N370S and V394L). Administration of IFG (30 mg/kg/day) to the mice homozygous for GCase mutations (V394L, D409H, or D409V) led to increased GCase activity in visceral tissues and brain extracts.