[Translation] A Phase III, Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Study of Ataluren and Open-label Extension in Patients with Nonsense Mutant DMD
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 effect of Ataluren on walking and endurance as assessed by the six-minute walk test (6MWT). The secondary objectives of this study were to determine the effect of Ataluren on walking and explosive activity as assessed by timed functional testing; to determine the effect of Ataluren on lower extremity muscle function as assessed by the Polaris Movement Assessment Scale (NSAA); and to evaluate the safety profile of Ataluren.