While multiple therapeutic approaches aimed at decreasing GBE1 substrate levels by targeting glycogen synthase 1 [9–11], enhancing GBE1 enzyme activity [12], and promoting lysosomal degradation of PBs [13] are in preclinical and clinical development, there are currently no effective disease-modifying therapies available for APBD. This evidence concerns the gene GBE1 and adult polyglucosan body disease.