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Publication: Assessment of treatment effect with multiple outcomes in 2 clinical trials of patients with Duchenne muscular dystrophy (DMD)

This research letter, published in the JAMA Network Open, presents an analysis of 2 randomized, double-blind, placebo-controlled trials of a mutation-specific DMD gene therapy

A statistical analysis to evaluate the totality of evidence was conducted combing data across primary (6MWD) and secondary (timed function test [TFT]) endpoints from the phase 2b and phase 3 trials of a mutation-specific DMD therapy

▼ 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.

Registration conditions differ internationally; always consult local prescribing information and/or Summary of Product Characteristics before prescribing any products.

MED-ALL-ATLN-2500006 | September 2025

Li D, McDonald CM, Elfring GL, et al. JAMA Netw Open. 2020;3(2):e1921306

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MED-ALL-DMD-2200190 | June 2025

Publication: Assessment of treatment effect with multiple outcomes in 2 clinical trials of patients with Duchenne muscular dystrophy (DMD)

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