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

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 two 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

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

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