%0 Journal Article %T Editorial for focused issue “Pompe disease: from basics to current and emerging therapies” %A Puertollano, Rosa %A Raben, Nina %J Annals of Translational Medicine %D 2019 %B 2019 %9 %! Editorial for focused issue “Pompe disease: from basics to current and emerging therapies” %K %X 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. %U https://atm.amegroups.org/article/view/25979 %V 7 %N 13 %P 275 %@ 2305-5847