GAA and glycogen storage disease II: 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.