@article{923e0dbddd214664958314d117f9aede,
title = "Meta-analyses of ataluren randomized controlled trials in nonsense mutation Duchenne muscular dystrophy: Journal of Comparative Effectiveness Research",
abstract = "Aim: Assess the totality of efficacy evidence for ataluren in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD). Materials methods: Data from the two completed randomized controlled trials (ClinicalTrials.gov: NCT00592553; NCT01826487) of ataluren in nmDMD were combined to examine the intent-to-treat (ITT) populations and two patient subgroups (baseline 6-min walk distance [6MWD] ≥300-",
keywords = "6-minute walk distance, ataluren, Duchenne muscular dystrophy, efficacy, meta-analyses, nonsense mutation Duchenne muscular dystrophy, randomized controlled trials, clinical effectiveness, clinical trial (topic), disease exacerbation, drug effect, drug efficacy, drug response, drug safety, human, meta analysis, nonsense mutation, priority journal, Review, risk factor, six minute walk test, treatment outcome",
author = "C. Campbell and R.J. Barohn and E. Bertini and B. Chabrol and G.P. Comi and B.T. Darras and R.S. Finkel and K.M. Flanigan and N. Goemans and S.T. Iannaccone and K.J. Jones and J. Kirschner and J.K. Mah and K.D. Mathews and C.M. McDonald and E. Mercuri and Y. Nevo and Y. P{\'e}re{\'o}n and J.B. Renfroe and M.M. Ryan and J.B. Sampson and U. Schara and T. Sejersen and K. Selby and M. Tulinius and J.J. V{\'i}lchez and T. Voit and L.-J. Wei and B.L. Wong and G. Elfring and M. Souza and J. McIntosh and P. Trifillis and S.W. Peltz and F. Muntoni",
note = "Cited By :3 Export Date: 19 March 2021 Correspondence Address: Campbell, C.; Schulich School of Medicine and Dentistry, Canada; email: craig.campbell@lhsc.on.ca",
year = "2020",
doi = "10.2217/cer-2020-0095",
language = "English",
volume = "9",
pages = "973--984",
journal = "J. Comp. Eff. Res.",
issn = "2042-6305",
publisher = "Future Medicine Ltd.",
number = "14",
}