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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 2b trial and a phase 3 trial (Ataluren Confirmatory Trial in DMD [ACT DMD])
Disease progression, as measured by 6-minute walk test (6MWT) and timed function tests (TFTs), in 2 prespecified subgroups and ITT population are discussed

▼ Ataluren is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions via the national reporting system. Adverse events should also be reported to PTC Therapeutics at pharmacovigilance@ptcbio.com.

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MED-ALL-ATLN-2500006 | September 2025

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

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MED-ALL-DMD-2200190 | June 2025

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

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