The current standard of care for Pompe disease is enzyme replacement therapy (ERT), comprising repeated intravenous infusions of a recombinant human GAA (rhGAA) precursor enzyme (alglucosidase alfa, avalglucosidase alfa, or cipaglucosidase alfa with miglustat), which were first approved in 2006, 2022, and 2023, respectively. This evidence concerns the gene GAA and Glycogen storage disease due to acid maltase deficiency.