None of these differences were significant; there was a numerical trend to a slightly larger loss (by 1.2 units in the CIDD) during deflazacort treatment compared to placebo (Figure 3 a; change between start and end of pooled crossover periods: deflazacort: d = −2.24; standard deviation (SD) = 3.0; placebo: d = −1.00, SD = 2.2; mixed model result (ITT): p(treatment) = 0.1558; 95% confidence interval [−3.01; 0.52] in disfavor of deflazacort; non-parametric analysis (PP): p = 0.4375).
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Treatment of dysferlinopathy with deflazacort: a double-blind, placebo-controlled clinical trial.
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