Efficacy and Safety of Vamorolone Over 48 Weeks in Boys With Duchenne Muscular Dystrophy: A Randomized Controlled Trial
Vamorolone治疗杜氏肌营养不良症男孩48周的疗效和安全性:一项随机对照试验
期刊:Neurology
影响因子:8.5
doi:10.1212/WNL.0000000000208112
Dang, Utkarsh J; Damsker, Jesse M; Guglieri, Michela; Clemens, Paula R; Perlman, Seth J; Smith, Edward C; Horrocks, Iain; Finkel, Richard S; Mah, Jean K; Deconinck, Nicolas; Goemans, Nathalie M; Haberlová, Jana; Straub, Volker; Mengle-Gaw, Laurel; Schwartz, Benjamin D; Harper, Amy; Shieh, Perry B; De Waele, Liesbeth; Castro, Diana; Yang, Michele L; Ryan, Monique M; McDonald, Craig M; Tulinius, Mar; Webster, Richard I; Mcmillan, Hugh J; Kuntz, Nancy; Rao, Vamshi K; Baranello, Giovanni; Spinty, Stefan; Childs, Anne-Marie; Sbrocchi, Annie M; Selby, Kathryn A; Monduy, Migvis; Nevo, Yoram; Vilchez, Juan J; Nascimento-Osorio, Andres; Niks, Erik H; De Groot, Imelda J M; Katsalouli, Marina; Van Den Anker, John N; Ward, Leanne M; Leinonen, Mika; D'Alessandro, Andrea L; Hoffman, Eric P