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Cipaglucosidase Alfa Plus Miglustat vs Alglucosidase Alfa Plus Placebo in Late-Onset Pompe Disease
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The Lancet Neurology
From MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine.
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
Lancet Neurol 2021 Dec 01;20(12)1027-1037, B Schoser, M Roberts, BJ Byrne, S Sitaraman, H Jiang, P Laforêt, A Toscano, J Castelli, J Díaz-Manera, M Goldman, AT van der Ploeg, D Bratkovic, S Kuchipudi, T Mozaffar, PS KishnaniFrom MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine.