Meta-analyses of ataluren randomized controlled trials in nonsense mutation Duchenne muscular dystrophy

Campbell C, Barohn RJ, Bertini E, Chabrol B, Comi GP, Darras BT, et al. Meta-analyses of ataluren randomized controlled trials in nonsense mutation Duchenne muscular dystrophy. Journal of comparative effectiveness research. 2020;9:973-984   Abstract Aim: Assess the totality of efficacy evidence for ataluren in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD). Materials & methods: Data from […]