Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial -

Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial
Information type: Randomised controlled trials
Source: The Lancet
Specialities: Neurological disorders
Summary
This RCT (n=230) reports no difference in the least-squares mean change in 6-minute walk distance (6MWD) from baseline to week 48 in ataluren vs placebo-treated patients (−47.7m for ataluren-treated patients and −60.7m for placebo-treated patients (difference 13.0m; p=0.213)).
UKMi comment
In a related editorial, the authors discuss the findings of this study, deliberating over the appropriateness of the use of the 6MWD, which is effort dependent and hence has restricted sensitivity in patients with higher baseline function, and shows substantial interpatient variability in patients with lower baseline function.
Related links: Editorial