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Meta-analyses of at...
Meta-analyses of ataluren randomized controlled trials in nonsense mutation Duchenne muscular dystrophy
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Campbell, C. (author)
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Barohn, R. J. (author)
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Bertini, E. (author)
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Chabrol, B. (author)
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Comi, G. P. (author)
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Darras, B. T. (author)
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Finkel, R. S. (author)
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Flanigan, K. M. (author)
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Goemans, N. (author)
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- Iannaccone, S. T. (author)
- Karolinska Institutet
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Jones, K. J. (author)
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Kirschner, J. (author)
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Mah, J. K. (author)
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Mathews, K. D. (author)
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McDonald, C. M. (author)
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Mercuri, E. (author)
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Nevo, Y. (author)
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Pereon, Y. (author)
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Ben Renfroe, J. (author)
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Ryan, M. M. (author)
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Sampson, J. B. (author)
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Schara, U. (author)
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Sejersen, T. (author)
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Selby, K. (author)
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- Tulinius, Mar, 1953 (author)
- Gothenburg University,Göteborgs universitet,Institutionen för kliniska vetenskaper,Institute of Clinical Sciences
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Vilchez, J. J. (author)
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Voit, T. (author)
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Wei, L. J. (author)
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Wong, B. L. (author)
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Elfring, G. (author)
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Souza, M. (author)
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McIntosh, J. (author)
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Trifillis, P. (author)
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Peltz, S. W. (author)
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Muntoni, F. (author)
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P. TC124-GD-007-Study Grp, P. TC124-GD-007-Study Grp (author)
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Act Dmd Study Grp, Act Dmd Study Grp (author)
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Clinical Evaluator Training, Grp (author)
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(creator_code:org_t)
- Becaris Publishing Limited, 2020
- 2020
- English.
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In: Journal of Comparative Effectiveness Research. - : Becaris Publishing Limited. - 2042-6305 .- 2042-6313. ; 9:14
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https://gup.ub.gu.se...
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Abstract
Subject headings
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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 (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.
Subject headings
- MEDICIN OCH HÄLSOVETENSKAP -- Annan medicin och hälsovetenskap (hsv//swe)
- MEDICAL AND HEALTH SCIENCES -- Other Medical and Health Sciences (hsv//eng)
Keyword
- 6-minute walk distance
- ataluren
- Duchenne muscular dystrophy
- efficacy
- meta-analyses
- nonsense mutation Duchenne muscular dystrophy
- randomized
- controlled trials
- 6-minute walk test
- end-points
- multicenter
- Health Care Sciences & Services
Publication and Content Type
- ref (subject category)
- art (subject category)
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To the university's database
- By the author/editor
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Campbell, C.
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Barohn, R. J.
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Bertini, E.
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Chabrol, B.
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Comi, G. P.
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Darras, B. T.
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show more...
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Finkel, R. S.
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Flanigan, K. M.
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Goemans, N.
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Iannaccone, S. T ...
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Jones, K. J.
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Kirschner, J.
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Mah, J. K.
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Mathews, K. D.
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McDonald, C. M.
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Mercuri, E.
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Nevo, Y.
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Pereon, Y.
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Ben Renfroe, J.
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Ryan, M. M.
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Sampson, J. B.
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Schara, U.
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Sejersen, T.
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Selby, K.
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Tulinius, Mar, 1 ...
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Vilchez, J. J.
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Voit, T.
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Wei, L. J.
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Wong, B. L.
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Elfring, G.
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Souza, M.
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McIntosh, J.
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Trifillis, P.
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Peltz, S. W.
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Muntoni, F.
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P. TC124-GD-007- ...
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Act Dmd Study Gr ...
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Clinical Evaluat ...
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show less...
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- MEDICAL AND HEALTH SCIENCES
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MEDICAL AND HEAL ...
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Journal of Compa ...
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University of Gothenburg
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Karolinska Institutet