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

Title:	Switching treatment to cipaglucosidase alfa plus miglustat positively affects patient-reported outcome measures in patients with late-onset Pompe disease
Authors:	Kishnani, Priya S.^Aff1; Byrne, Barry J.^Aff2; Claeys, Kristl G.^{Aff3, Aff4, IDs4168702400805w_cor3}; Díaz-Manera, Jordi^{Aff5, Aff6, Aff7}; Dimachkie, Mazen M.^Aff8; Kushlaf, Hani^Aff9; Mozaffar, Tahseen^Aff10; Roberts, Mark^Aff11; Schoser, Benedikt^Aff12; Hummel, Noemi^Aff13; Kopiec, Agnieszka^Aff14; Holdbrook, Fred^Aff15; Shohet, Simon^Aff16; Toscano, Antonio^Aff17; 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
Source:	Journal of Patient-Reported Outcomes. 8(1)
Database:	Springer Nature Journals