M. James

Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial

Craig M. McDonald, Craig Campbell, Ricardo Erazo Torricelli et al. · The Lancet · 2017 · 446 citations · Full text

Rare Diseases, Urology, Clinical Trials +5

Muscle MRI in patients with dysferlinopathy: pattern recognition and implications for clinical trials

Jordi Díaz‐Manera, Roberto Fernández‐Torrón, Jaume Llauger et al. · Journal of Neurology Neurosurgery & Psychiatry · 2018 · 111 citations · Full text

The Clinical Outcome Study for dysferlinopathy

Elizabeth Harris, Catherine L. Bladen, Anna Mayhew et al. · Neurology Genetics · 2016 · 108 citations · Full text

Efficacy and Safety of Vamorolone vs Placebo and Prednisone Among Boys With Duchenne Muscular Dystrophy

Michela Guglieri, Paula R. Clemens, Seth J. Perlman et al. · JAMA Neurology · 2022 · 107 citations · Full text

Performance of Upper Limb module for Duchenne muscular dystrophy

Anna Mayhew, Giorgia Coratti, Elena Mazzone et al. · Developmental Medicine & Child Neurology · 2019 · 70 citations · Full text