National Center for Tuberculosis and Lung Disease

Ambroxol in Gaucher disease and GBA1-related Parkinson disease: an updated systematic review

Abstract Background Ambroxol (ABX) is a central nervous system (CNS)-penetrant compound that can increase glucocerebrosidase activity and has been investigated in Gaucher disease (GD) and GBA1 -related Parkinson disease ( GBA1 -PD). Methods This PRISMA 2020 systematic review and...