Confirmatory long-term efficacy and safety results of ataluren in patients with nmDMD from Study 041, an international, randomized, double-blind, placebo-controlled, Phase III trial
来自 041 研究的确认性长期疗效和安全性结果，该研究是一项国际性、随机、双盲、安慰剂对照的 III 期试验，旨在评估阿塔卢伦治疗非转移性杜氏肌营养不良症 (nmDMD) 患者的疗效和安全性。
期刊:Journal of Comparative Effectiveness Research
影响因子:2.5
doi:10.57264/cer-2024-0238
Vlodavets, Dmitry; Wu, Shiwen; Kostera-Pruszczyk, Anna; Chae, Jong-Hee; Gulati, Sheffali; Shin, Jin-Hong; Lorentzos, Michelle; Cairns, Anita; Jong, Yuh-Jyh; Komaki, Hirofumi; Statland, Jeffrey; de Queiroz Campos Araujo, Alexandra Prufer; Gurgel-Giannetti, Juliana; Takeshima, Yasuhiro; Escobar Cedillo, Rosa E; Karachunski, Peter; Haginoya, Kazuhiro; Penematsa, Vinay; Chou, Connie; Williams, Paula; Werner, Christian; McDonald, Craig M
