[Translation] A Phase III, randomized, double-blind, placebo-controlled efficacy and safety study of Ataluren in patients with nonsense mutation DMD and an open-label extension study
本研究的主要目的是通过六分钟步行试验(6MWT)评估确定Ataluren 对行走和耐力的影响。本研究的次要目的为,通过计时功能测试评估确定Ataluren 对行走和爆发式活动的影响;通过北极星移动评价量表(NSAA)评估确定Ataluren 对下肢肌肉功能的影 响;评价Ataluren 的安全性特征。
[Translation] The primary objective of this study was to determine the effects of Ataluren on walking and endurance as assessed by the Six-Minute Walk Test (6MWT). Secondary objectives of this study were to determine the effects of Ataluren on walking and burst activity as assessed by the Timed Functional Test; to determine the effects of Ataluren on lower extremity muscle function as assessed by the North Star Ambulatory Assessment (NSAA); and to evaluate the safety profile of Ataluren.