Following years of drug development, preclinical studies in a mouse model [22,23], and two pivotal clinical trials [24,25], recombinant human GAA-a drug called alglucosidase alfa (rhGAA; Myozyme© (ex-US) and Lumizyme© (US); Genzyme, Cambridge, Massachusetts)-received broad-label marketing approval for the treatment of Pompe disease in 2006. This evidence concerns the gene GAA and Glycogen storage disease due to acid maltase deficiency.