학술논문
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
Document Type
Article
Author
Sebok, Agnes; Pestronk, Alan; Dominovic-Kovacevic, Aleksandra; Khan, Aneal; Koritnik, Blaž; Tard, Celine; Lindberg, Christopher; Quinn, Colin; Kornblum, Cornelia; Eldridge, Crystal; Bodkin, Cynthia; Reyes-Leiva, David; Hughes, Derralynn; Stefanescu, Ela; SALORT-CAMPANA, Emmanuelle; Butler, Ernest; Bouhour, Francoise; Kim, Gee; Konstantinos Papadimas, George; 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; Cauci, Jonathan; Alonso-Pérez, Jorge; Janszky, Jozsef; Berthy, Julie; Gutschmidt, Kristina; Claeys, Kristl; Judit Molnar, Maria; Wencel, Marie; Tarnopolsky, Mark; Dimachkie, Mazen; 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; Schoser, Benedikt ; Roberts, Mark ; Byrne, Barry J; Sitaraman, Sheela; Jiang, Hai; Laforêt, Pascal; Toscano, Antonio; Castelli, Jeff; Díaz-Manera, Jordi; Goldman, Mitchell; van der Ploeg, Ans T; Bratkovic, Drago; Kuchipudi, Srilakshmi; Mozaffar, Tahseen ; Kishnani, Priya S
Source
In The Lancet Neurology December 2021 20(12):1027-1037
Subject
Language
ISSN
1474-4422