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)

2024 . Schoser, Benedikt | Kishnani, Priya S. | Bratkovic, Drago | Byrne, Barry J. | Claeys, Kristl G. | Díaz-Manera, Jordi | Laforêt, Pascal | Roberts, Mark | Toscano, Antonio | van der Ploeg, Ans T. | Castelli, Jeff | Goldman, Mitchell | Holdbrook, Fred | Sitaraman Das, Sheela | Wasfi, Yasmine | Mozaffar, Tahseen | the ATB200-07 Study Group | 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