In this special issue of Annals of Translational Medicine, well distinguished clinicians, academic researchers, industry scientists, and patient advocates address recent advances and challenges in current and future therapies for Pompe disease, a prototypical lysosomal storage disorder. As is the case with many maladies, the history of Pompe disease, a severe neuromuscular disorder, is a reflection of the accelerating pace of basic science discoveries and the speed with which scientific knowledge is translated into medical progress.
