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Zeitschriftenartikel
Attarian, Shahram; Young, Peter; Brannagan, Thomas H.; Adams, David; Damme, Philip van; Thomas, Florian P.; Casanovas, Carlos; Tard, Celine; Walter, Maggie C.; Pereon, Yann; Walk, David; Stino, Amro; Visser, Marianne de; Verhamme, Camiel; Amato, Anthony; Carter, Gregory; Magy, Laurent; Statland, Jeffrey M. und Felice, Kevin
(2021):
A double-blind, placebo-controlled, randomized trial of PXT3003 for the treatment of Charcot-Marie-Tooth type 1A.
In: Orphanet Journal of Rare Diseases, Bd. 16, Nr. 1, 433
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