Switching treatment to cipaglucosidase alfa plus miglustat positively affects patient-reported outcome measures in patients with late-onset Pompe disease

2024 . Kishnani, Priya S. | Byrne, Barry J. | Claeys, Kristl | Díaz-Manera, Jordi | Dimachkie, Mazen M. | Kushlaf, Hani | Mozaffar, Tahseen | Roberts, Mark | Schoser, Benedikt | Hummel, Noemi | Kopiec, Agnieszka | Holdbrook, Fred | Shohet, Simon | Toscano, Antonio | on behalf of the PROPEL 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 | Akihiro, Hashiguchi | Lau, Heather | Pedro, Helio | Andersen, Henning | Amartino, Hernan | Shiraishi, Hideaki | Kobayashi, Hiroshi | Tarnev, Ivaylo | Vengoechea, Jaime | Avelar, Jennifer | Shin, Jin-Hong | Cauci, Jonathan | Alonso-Pérez, Jorge | Janszky, Jozsef | Berthy, Julie | Kornblum, Cornelia | Gutschmidt, Kristina | Molnar, Maria Judit | Wencel, Marie | Tarnopolsky, Mark | Tchan, Michel | Freimer, Miriam | Longo, Nicola | 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