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
Schoser, Benedikt; Roberts, Mark*; Byrne, Barry J; Sitaraman, Sheela; Jiang, Hai; Laforêt, Pascal; Toscano, Antonio; Castelli, Jeff; Díaz-Manera, Jordi ✉; Goldman, Mitchell; der Ploeg, Ans Tvan; Bratkovic, Drago; Kuchipudi, Srilakshmi; Mozaffar, Tahseen**; Kishnani, Priya S; Sebok, Agnes [Sebők, Ágnes (Sejtbiológia), Collaborator]; Pestronk, Alan [Collaborator]; Dominovic-Kovacevic, Aleksandra [Collaborator]; ROPEL Study Group [Collaborative Organization]; Khan, Aneal [Collaborator]; Koritnik, Blaž [Collaborator]; Tard, Celine [Collaborator]; Lindberg, Christopher [Collaborator]; Quinn, Colin [Collaborator]; Eldridge, Crystal [Collaborator]; Bodkin, Cynthia [Collaborator]; Reyes-Leiva, David [Collaborator]; Hughes, Derralynn [Collaborator]; Stefanescu, Ela [Collaborator]; Salort-Campana, Emmanuelle [Collaborator]; Butler, Ernest [Collaborator]; Bouhour, Francoise [Collaborator]; Kim, Gee [Collaborator]; Papadimas, George Konstantinos [Collaborator]; Parenti, Giancarlo [Collaborator]; Bartosik-Psujek, Halina [Collaborator]; Hani, Kushlaf [Collaborator]; Hashiguchi, Akihiro [Collaborator]; Lau, Heather [Collaborator]; Pedro, Helio [Collaborator]; Andersen, Henning [Collaborator]; Amartino, Hernan [Collaborator]; Shiraishi, Hideaki [Collaborator]; Kobayashi, Hiroshi [Collaborator]; Tarnev, Ivaylo [Collaborator]; Vengoechea, Jaime [Collaborator]; Avelar, Jennifer [Collaborator]; Shin, Jin-Hong [Collaborator]; Cauci, Jonathan [Collaborator]; Alonso-Pérez, Jorge [Collaborator]; Janszky, Jozsef [Janszky, József Vladimír (Neurológia), Collaborator] Medical School
(UP); Berthy, Julie [Collaborator]; Kornblum, Cornelia [Collaborator]; Gutschmidt, Kristina [Collaborator]; Claeys, Kristl [Collaborator]; Molnar, Maria Judit [Molnár, Mária Judit (Neurológia), Collaborator] Genomic Medicine
and the Institute of Rare Dise... (SU / FM / I); Wencel, Marie [Collaborator]; Tarnopolsky, Mark [Collaborator]; Dimachkie, Mazen [Collaborator]; Tchan, Michel [Collaborator]; Freimer, Miriam [Collaborator]; Longo, Nicola [Collaborator]; Vidal-Fernandez, Nuria [Collaborator]; Musumeci, Olimpia [Collaborator]; Goker-Alpan, Ozlem [Collaborator]; Deegan, Patrick [Collaborator]; Clemens, R Paula [Collaborator]; Roxburgh, Richard [Collaborator]; Henderson, Robert [Collaborator]; Hopkin, Robert [Collaborator]; Sacconi, Sabrina [Collaborator]; Fecarotta, Simona [Collaborator]; Attarian, Shahram [Collaborator]; Wenninger, Stephan [Collaborator]; Dearmey, Stephanie [Collaborator]; Hiwot, Tarekegn [Collaborator]; Burrow, Thomas [Collaborator]; Ruck, Tobias [Collaborator]; Sawada, Tomo [Collaborator]; Vescei, Laszlo [Collaborator]; Löscher, Wolfgang [Collaborator]; Chien, Yin-Hsiu [Collaborator]
