Publication: Meta-analysis of ataluren randomized controlled trials in nonsense mutation Duchenne muscular dystrophy (nmDMD)
This research article, published in the Journal ofComparative 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
Campbell C, Barohn RJ, Bertini E, et al. J Comp Eff Res. 2020;9(14);973–9841
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▼ 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 at firstname.lastname@example.org.
Registration conditions differ internationally, and prescribing information may vary depending on local approval in each country. The European Summary of Product Characteristics for ataluren is available here. Please refer to your local country guidance for more information.
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MED-ALL-DMD-2200190 | August 2022
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