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Meta-analyses of ataluren randomized controlled trials in nonsense mutation Duchenne muscular dystrophy.
  • Craig Campbell
  • Richard J Barohn
  • Enrico Bertini
  • Brigitte Chabrol
  • Giacomo Pietro Comi
  • Basil T Darras
  • Richard S Finkel
  • Kevin M Flanigan
  • Nathalie Goemans
  • Susan T Iannaccone
  • Kristi J Jones
  • Janbernd Kirschner
  • Jean K Mah
  • Katherine D Mathews
  • Craig M McDonald
  • Eugenio Mercuri
  • Yoram Nevo
  • Yann Péréon
  • J Ben Renfroe
  • Monique M Ryan
  • Jacinda B Sampson
  • Ulrike Schara
  • Thomas Sejersen
  • Kathryn Selby
  • Már Tulinius
  • Juan J Vílchez
  • Thomas Voit
  • Lee-Jen Wei
  • Brenda L Wong
  • Gary Elfring
  • Marcio Souza
  • Joseph McIntosh
  • Panayiota Trifillis
  • Stuart W Peltz
  • Francesco Muntoni
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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 ( 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.

Citation Information
Craig Campbell, Richard J Barohn, Enrico Bertini, Brigitte Chabrol, et al.. "Meta-analyses of ataluren randomized controlled trials in nonsense mutation Duchenne muscular dystrophy." JOURNAL OF COMPARATIVE EFFECTIVENESS RESEARCH Vol. 9 Iss. 14 (2020) p. 973 - 984
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