1. The six boys treated with 30 mg and 50 mg of eteplirsen from the beginning of the study have demonstrated remarkable stability in walking performance. After 96 weeks, the distance walked over six minutes has declined by just 6.3 meters compared to baseline.
The 96-week data are no fluke: Here are the changes in six-minute walk distance from baseline for the eteplirsen-treated boys from weeks 74, 84 and 96: -2.2 meters, -9.2 meters, -6.3 meters.
2. The four boys in the study initially treated with a placebo and switched to eteplirsen at week 25 (the crossover group) are also showing stable walking ability now that the effect of the drug has kicked in.The best way to see the benefit of eteplirsen in these patients is to compare six-minute walk test performance between week 74 and week 96. At week 74, these four boys walked 62.4 meters less than at baseline. At week 96, the drop in walking distance was 67.7 meters. Basically the same. Why skip the comparison with the week 84 measurement? Because one of the boys in the crossover group broke his ankle and was unable to perform the six-minute walk test for the week 84 analysis, thereby skewing the mean walking performance higher.
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