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104-week efficacy and safety of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease: a phase III open-label extension study (ATB200-07)

Title: 104-week efficacy and safety of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease: a phase III open-label extension study (ATB200-07)
Authors: Schoser, BenediktAff1, IDs00415024122360_cor1; Kishnani, Priya S.Aff2; Bratkovic, DragoAff3; Byrne, Barry J.Aff4; Claeys, Kristl G.Aff5, Aff6; Díaz-Manera, JordiAff7; Laforêt, PascalAff8; Roberts, MarkAff9; Toscano, AntonioAff10; van der Ploeg, Ans T.Aff11; Castelli, JeffAff12; Goldman, MitchellAff12; Holdbrook, FredAff12; Sitaraman Das, SheelaAff12; Wasfi, YasmineAff12; Mozaffar, TahseenAff13; Sebok, Agnes; Pestronk, Alan; Dominovic-Kovacevic, Aleksandra; Khan, Aneal; Koritnik, Blaž; Tard, Celine; Lindberg, Christopher; Quinn, Colin; Eldridge, Crystal; Bodkin, Cynthia; Reyes-Leiva, David; Hughes, Derralynn; Stefanescu, Ela; Salort-Campana, Emmanuelle; Butler, Ernest; Bouhour, Francoise; Kim, Gee; Papadimas, George Konstantinos; Parenti, Giancarlo; Bartosik-Psujek, Halina; Kushlaf, Hani; Akihiro, Hashiguchi; Lau, Heather; Pedro, Helio; Andersen, Henning; Amartino, Hernan; Shiraishi, Hideaki; Kobayashi, Hiroshi; Tarnev, Ivaylo; Vengoechea, Jaime; Avelar, Jennifer; Shin, Jin-Hong; Nevin, John; Cauci, Jonathan; Alonso-Pérez, Jorge; Janszky, Jozsef; Berthy, Julie; Kornblum, Cornelia; Gutschmidt, Kristina; Molnar, Maria Judit; Wencel, Marie; Tarnopolsky, Mark; Boentert, Matthias; Tchan, Michel; Freimer, Miriam; Longo, Nicola; Abreu, Nicolas; Vidal-Fernandez, Nuria; Musumeci, Olimpia; Goker-Alpan, Ozlem; Deegan, Patrick; Clemens, Paula R.; Roxburgh, Richard; Henderson, Robert; Hopkin, Robert; Sacconi, Sabrina; Fecarotta, Simona; Attarian, Shahram; Wenninger, Stephan; Dearmey, Stephanie; Hiwot, Tarekegn; Burrow, Thomas; Ruck, Tobias; Sawada, Tomo; Laszlo, Vescei; Löscher, Wolfgang; Chien, Yin-Hsiu
Source: Journal of Neurology. 271(5):2810-2823
Database: Springer Nature Journals