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Zeitschriftenartikel
Diaz-Manera, Jordi; Kishnani, Priya S.; Kushlaf, Hani; Ladha, Shafeeq; Mozaffar, Tahseen; Straub, Volker; Toscano, Antonio; Ploeg, Ans T. van der; Berger, Kenneth I.; Clemens, Paula R.; Chien, Yin-Hsiu; Day, John W.; Illarioshkin, Sergey; Roberts, Mark; Attarian, Shahram; Borges, Joao Lindolfo; Bouhour, Francoise; Choi, Young Chul; Erdem-Ozdamar, Sevim; Goker-Alpan, Ozlem; Kostera-Pruszczyk, Anna; Haack, Kristina An; Hug, Christopher; Huynh-Ba, Olivier; Johnson, Judith; Thibault, Nathan; Zhou, Tianyue; Dimachkie, Mazen M. und Schoser, Benedikt
(2021):
Safety and efficacy of avalglucosidase alfa versus alglucosidase alfa in patients with late-onset Pompe disease (COMET): a phase 3, randomised, multicentre trial.
In: Lancet Neurology, Bd. 20, Nr. 12: S. 1012-1026
Schoser, Benedikt; Roberts, Mark; Byrne, Barry J.; Sitaraman, Sheela; Jiang, Hai; Laforet, Pascal; Toscano, Antonio; Castelli, Jeff; Diaz-Manera, Jordi; Goldman, Mitchell; Ploeg, Ans T. van der; Bratkovic, Drago; Kuchipudi, Srilakshmi; Mozaffar, Tahseen und Kishnani, Priya S.
(2021):
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.
In: Lancet Neurology, Bd. 20, Nr. 12: S. 1027-1037
Ploeg, Ans van der; Carlier, Pierre G.; Carlier, Robert-Yves; Kissel, John T.; Schoser, Benedikt; Wenninger, Stephan; Pestronk, Alan; Barohn, Richard J.; Dimachkie, Mazen M.; Goker-Alpan, Ozlem; Mozaffar, Tahseen; Pena, Loren D. M.; Simmons, Zachary; Straub, Volker; Guglieri, Michela; Young, Peter; Boentert, Matthias; Baudin, Pierre-Yves; Wens, Stephan; Shafi, Raheel; Bjartmar, Carl und Thurberg, Beth L.
(2016):
Prospective exploratory muscle biopsy, imaging, and functional assessment in patients with late-onset Pompe disease treated with alglucosidase alfa: The EMBASSY Study.
In: Molecular Genetics and Metabolism, Bd. 119, Nr. 1-2: S. 115-123