duvoglustat + alglucosidase alfa

**Duvoglustat + alglucosidase alfa** is an investigational combination therapy for the treatment of Pompe disease (glycogen storage disease type II), a rare lysosomal storage disorder. The combination consists of duvoglustat, an oral small molecule inhibitor of acid alpha-glucosidase (GAA)-specific glycogen debranching enzymes, and alglucosidase alfa, a recombinant enzyme replacement therapy that provides exogenous acid alpha-glucosidase. The aim of combining duvoglustat with alglucosidase alfa is to enhance the effectiveness of enzyme replacement therapy by increasing the exposure, uptake, or activity of alglucosidase alfa in lysosomes, thereby improving glycogen clearance. Duvoglustat is being developed as an adjunct to alglucosidase alfa to improve the bioavailability and efficacy of enzyme replacement therapy.