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

Authors :: Campbell, C
Barohn, RJ
Bertini, E
Chabrol, B
Comi, GP
Darras, BT
Finkel, RS
Flanigan, KM
Goemans, N
Iannaccone, ST
Jones, KJ
Kirschner, J
Mah, JK
Mathews, KD
McDonald, CM
Mercuri, E
Nevo, Y
Pereon, Y
Ben Renfroe, J
Ryan, MM
Sampson, JB
Schara, U
Sejersen, T
Selby, K
Tulinius, M
VILCHEZ, JJ
Voit, T
Wei, LJ
Wong, BL
Elfring, G
Souza, M
McIntosh, J
Trifillis, P
Peltz, SW
Muntoni, F
PTC124-GD-007-Study Grp
ACT DMD Study Grp
Clinical Evaluator Training Grp
Source :: Journal of Comparative Effectiveness Research, r-IIS La Fe. Repositorio Institucional de Producción Científica del Instituto de Investigación Sanitaria La Fe, instname
Publication Year :: 2020
Publisher :: Future Medicine Ltd., 2020.
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-= 300-= 300

Subjects :: Duchenne muscular dystrophy
6-minute walk distance
nonsense mutation Duchenne muscular dystrophy
meta-analyses
efficacy
randomized controlled trials
ataluren

ISSN :: 20426305
Database :: OpenAIRE
Journal :: Journal of Comparative Effectiveness Research, r-IIS La Fe. Repositorio Institucional de Producción Científica del Instituto de Investigación Sanitaria La Fe, instname
Accession number :: edsair.RECOLECTA.....edd110e20c23be981bc90e66cf3ae59c

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