Table 1.

Current/Proposed therapeutic strategies targeting GCase

Therapeutic Strategy	Example	Phase in drug development	Summary of Results	GCase Activity Measurement Technique
Molecular Chaperone	Ambroxol	Phase II Completed	Decreased CSF GCase activity and increased protein levels	4-MU1 in vitro
Activator	BIA 28–6156/LTI-291	Phase I Completed	Effects on GCase activity not publicly disclosed	N/A
Gene therapy	PR001	Phase I/II Ongoing	CSF GCase activity increased from undetectable to within normal range	N/A
Transport Vehicle Modified Recombinant GCase	ETV:GBA2	Preclinical Research Ongoing	No current publications	N/A

¹4-MU (4-methylumbelliferyl-β-D-glucopyranoside)

²ETV (Enzyme Transport Vehicle)