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Publication: Meta-analysis of ataluren randomized controlled trials in nonsense mutation Duchenne muscular dystrophy (nmDMD)

This research article, published in the Journal of Comparative Effectiveness Research, examines the totality of efficacy evidence for a mutation-specific therapy in patients with nmDMD by meta-analysis

  • ​Review the outcomes of meta-analyses using the overall intent-to-treat (ITT) nmDMD populations of a phase IIb trial and a phase III trial (Ataluren Confirmatory Trial in DMD [ACT DMD])
  • Disease progression, as measured by 6-minute walk test (6MWT) and timed function tests (TFTs), in two prespecified subgroups and ITT population are discussed

Campbell C, Barohn RJ, Bertini E, et al. J Comp Eff Res.  2020;9(14);973–9841

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