The Avalglucosidase Alfa Phase 3 COMET Trial in Participants With Late-Onset Pompe Disease: Efficacy and Safety Results After 97 Weeks

Author(s):

Hani A. Kushlaf, MD, FAAN, FANA, FAANEM, University of Cincinnati, Cincinnati, OH, USA, Priya Kishnani, MD, Duke University Medical Center, Durham, NC, USA, Jordi Díaz-Manera, MD, Hospital de la Santa Cruz, Barcelona Spain, Shafeeq Ladha, MD, Gregory W. Fulton ALS and Neuromuscular Center, Barrow Neurological Institute, Phoenix, AZ, USA, Tahseen Mozaffar, MD, University of California, Irvine, CA, USA, Volker Straub, MD, PhD, John Walton Muscular Dystrophy Research Centre and Newcastle Hospitals NHS Foundation Trust, UK, Antonio Toscano, MD, Reference Center for Rare Neuromuscular Disorders, University of Messina, Messina, Italy, Ans T. van der Ploeg, MD, PhD, Erasmus MC University Medical Center, Rotterdam, Netherlands, Kenneth I. Berger, MD, NYU Grossman School of Med and André Cournand Pulmonary Physiology Laboratory, New York, USA, Paula R. Clemens, MD, University of Pittsburgh School of Medicine, VA Pittsburgh Healthcare System, Pittsburgh, PA, USA, Yin-Hsiu Chien, MD, Department of Medical Genetics and Pediatrics, National Taiwan University Hospital, Taipei, Taiwan, John Day, MD, PhD, Stanford University, Sergey Illarioshkin, MD, Research Center of Neurology, Moscow, Russia, Mark Roberts, MD, PhD, Salford Royal NHS Foundation Trust, Salford, UK, Shahram Attarian, MD, Referral Centre for Neuromuscular Diseases and ALS, Hôpital La Timone, Marseille, France, Gerson Carvalho, MD, Instituto Chronos de Apoio à Pesquisa, Brasília, DF, Brazil, Young Chul Choi, MD, Gangnam Severance Hospital, Yonsei University, College of Medicine, Seoul, Korea, Sevim Erdem-Özdamar, MD, Department of Neurology, Hacettepe University Faculty of Medicine, Ankara, Turkey, Ozlem Goker-Alpan, MD, Lysosomal and Rare Disorders Research and Treatment Center, Fairfax, VA, USA, Anna Kostera-Pruszczyk, MD, PhD, Department of Neurology, Medical University of Warsaw, Warsaw, Poland, Kristina An Haack, MD, Sanofi Genzyme, Chilly-Mazarin, France, Olivier Huynh-Ba, MD, Sanofi Genzyme, Chilly-Mazarin, France, Swathi Tammireddy, MBBS, Sanofi Genzyme, Cambridge, MA, USA, Nathan Thibault, PharmD, Sanofi Genzyme, Cambridge, MA, USA, Tianyue Zhou, PhD, Sanofi Genzyme, Cambridge, MA, USA, Mazen M Dimachkie, MD, University of Kansas Medical Center, Department of Neurology, Kansas City, KS, USA, Benedikt Schoser, MD, PhD, Friedrich-Baur-Institut, Ludwig-Maximilians-Universität München, Munich, Germany