Cipaglucosidase Alfa-Miglustat Versus Alglucosidase Alfa-Placebo in Late-Onset Pompe Disease (LOPD): PROPEL Study Subgroup Analyses

Pompe disease is a rare autosomal recessive disorder characterized by progressive loss of muscle and respiratory function due to acid α-glucosidase (GAA) deficiency. The approved treatment is enzyme-replacement therapy (ERT) with recombinant human GAA (rhGAA) alglucosidase alfa. Cipaglucosidase alfa/miglustat is an investigational, two-component therapy comprising cipaglucosidase alfa, a novel rhGAA with enhanced glycosylation for improved uptake and processing, and miglustat, an enzyme stabilizer.