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

Author:

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,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,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,Cornelia Kornblum,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

Publisher

Elsevier BV

Subject

Clinical Neurology

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