Loren D.M. Pena, MD, PhD, Associate Professor of Pediatrics at the University of Cincinnati College of Medicine, Cincinnati Children’s Hospital, Cincinnati, Ohio, discusses treatment advances in Pompe disease. Enzyme replacement therapy has provided a breakthrough in the management of Pompe disease; however, Dr Pena notes that unmet medical needs remain. Next-generation enzyme replacement therapy may have a greater therapeutic effect due to improved targeting. According to Dr Pena, gene therapy has the potential to change the treatment of Pompe disease.