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  • Campbell, C. (author)

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

  • Article/chapterEnglish2020

Publisher, publication year, extent ...

  • Becaris Publishing Limited,2020

Numbers

  • LIBRIS-ID:oai:gup.ub.gu.se/297433
  • https://gup.ub.gu.se/publication/297433URI
  • https://doi.org/10.2217/cer-2020-0095DOI
  • http://kipublications.ki.se/Default.aspx?queryparsed=id:144520616URI

Supplementary language notes

  • Language:English

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  • Subject category:ref swepub-contenttype
  • Subject category:art swepub-publicationtype

Notes

  • 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-<400 or <400 m). Meta-analyses examined 6MWD change from baseline to week 48.Results:Statistically significant differences in 6MWD change with ataluren versus placebo were observed across all three meta-analyses. Least-squares mean difference (95% CI): ITT (n = 342), +17.2 (0.2-34.1) m, p = 0.0473; >= 300-<400 m (n = 143), +43.9 (18.2-69.6) m, p = 0.0008; <400 m (n = 216), +27.7 (6.4-49.0) m, p = 0.0109.Conclusion:These meta-analyses support previous evidence for ataluren in slowing disease progression versus placebo in patients with nmDMD over 48 weeks. Treatment benefit was most evident in patients with a baseline 6MWD >= 300-<400 m (the ambulatory transition phase), thereby informing future trial design.

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  • Barohn, R. J. (author)
  • Bertini, E. (author)
  • Chabrol, B. (author)
  • Comi, G. P. (author)
  • Darras, B. T. (author)
  • Finkel, R. S. (author)
  • Flanigan, K. M. (author)
  • Goemans, N. (author)
  • Iannaccone, S. T.Karolinska Institutet (author)
  • Jones, K. J. (author)
  • Kirschner, J. (author)
  • Mah, J. K. (author)
  • Mathews, K. D. (author)
  • McDonald, C. M. (author)
  • Mercuri, E. (author)
  • Nevo, Y. (author)
  • Pereon, Y. (author)
  • Ben Renfroe, J. (author)
  • Ryan, M. M. (author)
  • Sampson, J. B. (author)
  • Schara, U. (author)
  • Sejersen, T. (author)
  • Selby, K. (author)
  • Tulinius, Mar,1953Gothenburg University,Göteborgs universitet,Institutionen för kliniska vetenskaper,Institute of Clinical Sciences(Swepub:gu)xtulim (author)
  • Vilchez, J. J. (author)
  • Voit, T. (author)
  • Wei, L. J. (author)
  • Wong, B. L. (author)
  • Elfring, G. (author)
  • Souza, M. (author)
  • McIntosh, J. (author)
  • Trifillis, P. (author)
  • Peltz, S. W. (author)
  • Muntoni, F. (author)
  • P. TC124-GD-007-Study Grp, P. TC124-GD-007-Study Grp (author)
  • Act Dmd Study Grp, Act Dmd Study Grp (author)
  • Clinical Evaluator Training, Grp (author)
  • Karolinska InstitutetInstitutionen för kliniska vetenskaper (creator_code:org_t)

Related titles

  • In:Journal of Comparative Effectiveness Research: Becaris Publishing Limited9:142042-63052042-6313

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