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Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial

Author / Creator:Mercuri, Eugenio ; Vilchez, Juan J ; Boespflug-Tanguy, Odile ; Zaidman, Craig M ; Mah, Jean K ; Goemans, Nathalie ; Müller-Felber, Wolfgang ; Niks, Erik H ; Schara-Schmidt, Ulrike ; Bertini, Enrico ; Comi, Giacomo P ; Mathews, Katherine D ; Servais, Laurent ; Vandenborne, Krista ; Johannsen, Jessika ; Messina, Sonia ; Spinty, Stefan ; McAdam, Laura ; Selby, Kathryn ; Byrne, Barry ; Laverty, Chamindra G ; Carroll, Kevin ; Zardi, Giulia ; Cazzaniga, Sara ; Coceani, Nicoletta ; Bettica, Paolo ; McDonald, Craig M ; Acsadi, Gyula ; Baranello, Giovanni ; Blaschek, Astrid ; Brandsema, John ; Brogna, Claudia ; Bruno, Claudio ; Connolly, Anne ; de Groot, Imelda ; De Waele, Liesbeth ; Finanger, Erika ; Finkel, Richard ; Gidaro, Teresa ; Guglieri, Michaela ; Harper, Amy ; Lopez Lobato, Mercedes ; Madruga Garrido, Marcos ; Magri, Francesca ; Manousakis, Georgios ; Masson, Riccardo ; Monduy, Migvis ; Muelas Gomez, Nuria ; Munell, Francina ; Nascimento, Andres ; Nevo, Yoram ; Pereon, Yann ; Phan, Han ; Sansone, Valeria ; Scoto, Mariacristina ; Vucinic, Dragana ; Willis, Tracey

Publisher:England: Elsevier Ltd

Journal title:Lancet neurology, 2024-04, Vol.23 (4), p.393-403

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Relationship between MAN2B1 genotype/subcellular localization subgroups, antidrug antibody detection, and long‐term velmanase alfa treatment outcomes in patients with alpha‐mannosidosis

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Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial

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