Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease (PROPEL): an international, randomised, double-blind, parallel-group, phase 3 trial

Benedikt Schoser, Mark Roberts, Barry J. Byrne, Sheela Sitaraman, Hai Jiang, Pascal Laforêt, António Toscano, Jeff Castelli, Jordi Díaz‐Manera, Mitchell Goldman, Ans T. van der Ploeg, Drago Bratkovic, Srilakshmi Kuchipudi, Tahseen Mozaffar, Priya S. Kishnani, Ágnes Sebök, Alan Pestronk, Aleksandra Dominović-Kovačević, Aneal Khan, Blaž Koritnik, Céline Tard, Christopher Lindberg, Colin Quinn, Cornelia Kornblum, Crystal Eldridge, Cynthia Bodkin, David Reyes‐Leiva, Derralynn Hughes, Ela Stefanescu, Emmanuelle Salort‐Campana, Ernest Butler, Françoise Bouhour, Gee Kim, George K. Papadimas, Giancarlo Parenti, Halina Bartosik-Psujek, Hani Kushlaf, Akihiro Hashiguchi, Heather Lau, Hélio Pedro, Henning Andersen, Hernán Amartino, Hideaki Shiraishi, Hiroshi Kobayashi, Ivaylo Tarnev, Jaime Vengoechea, Jennifer Avelar, Jin‐Hong Shin, Jonathan Cauci, Jorge Alonso‐Pérez, József Janszky, Julie Berthy, Kristina Gutschmidt, Kristl G. Claeys, Mária Judit Molnár, Marie Wencel, Mark A. Tarnopolsky, Mazen M. Dimachkie, Michel Tchan, Miriam Freimer, Nicola Longo, Nuria Vidal-Fernandez, Olimpia Musumeci, Özlem Göker-Alpan, Patrick Deegan, Paula R. Clemens, Richard Roxburgh, Robert Henderson, Robert J. Hopkin, Sabrina Sacconi, Simona Fecarotta, Shahram Attarian, Stephan Wenninger, Stephanie DeArmey, Tarekegn Hiwot, Thomas Andrew Burrow, Tobias Ruck, Tomo Sawada, Vescei Laszlo, Wolfgang N. Löscher, Yin‐Hsiu Chien