Meta-analyses of ataluren randomized controlled trials in nonsense mutation Duchenne muscular dystrophy: Journal of Comparative Effectiveness Research

C. Campbell, R.J. Barohn, E. Bertini, B. Chabrol, G.P. Comi, B.T. Darras, R.S. Finkel, K.M. Flanigan, N. Goemans, S.T. Iannaccone, K.J. Jones, J. Kirschner, J.K. Mah, K.D. Mathews, C.M. McDonald, E. Mercuri, Y. Nevo, Y. Péreón, J.B. Renfroe, M.M. RyanJ.B. Sampson, U. Schara, T. Sejersen, K. Selby, M. Tulinius, J.J. Vílchez, T. Voit, L.-J. Wei, B.L. Wong, G. Elfring, M. Souza, J. McIntosh, P. Trifillis, S.W. Peltz, F. Muntoni

Research output: Contribution to journalArticlepeer-review

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-
Original languageEnglish
Pages (from-to)973-984
Number of pages12
JournalJ. Comp. Eff. Res.
Volume9
Issue number14
DOIs
Publication statusPublished - 2020

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

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