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.

Efficacy end points in the ITT population Primary end point The difference in the average slope of change in 6MWD over 72 weeks between the ataluren and placebo groups was nominally significant (-0.74 m/week and -0.94 m/week in the ataluren and placebo groups, respectively; difference [95% CI]: 0.20 [0.03, 0.38]; p = 0.0248), equivalent to a 21% slower rate of decline in the ataluren group ( Table 3 ).

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This may also explain why results for the primary end point and several secondary end points in this subgroup numerically favored ataluren but did not reach statistical significance.

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