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

Autor:	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
Rok vydání:	2020
Předmět:	Duchenne muscular dystrophy 6-minute walk distance nonsense mutation Duchenne muscular dystrophy meta-analyses efficacy randomized controlled trials ataluren
Zdroj:	Journal of Comparative Effectiveness Research r-IIS La Fe. Repositorio Institucional de Producción Científica del Instituto de Investigación Sanitaria La Fe instname
ISSN:	2042-6305
Popis:	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
Databáze:	OpenAIRE
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