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

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

Assess the totality of efficacy evidence for ataluren in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD). 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...

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Veröffentlicht in:Journal of comparative effectiveness research 2020-10, Vol.9 (14), p.973-984
Hauptverfasser: Campbell, Craig, Barohn, Richard J, Bertini, Enrico, Chabrol, Brigitte, Comi, Giacomo Pietro, Darras, Basil T, Finkel, Richard S, Flanigan, Kevin M, Goemans, Nathalie, Iannaccone, Susan T, Jones, Kristi J, Kirschner, Janbernd, Mah, Jean K, Mathews, Katherine D, McDonald, Craig M, Mercuri, Eugenio, Nevo, Yoram, Péréon, Yann, Renfroe, J Ben, Ryan, Monique M, Sampson, Jacinda B, Schara, Ulrike, Sejersen, Thomas, Selby, Kathryn, Tulinius, Már, Vílchez, Juan J, Voit, Thomas, Wei, Lee-Jen, Wong, Brenda L, Elfring, Gary, Souza, Marcio, McIntosh, Joseph, Trifillis, Panayiota, Peltz, Stuart W, Muntoni, Francesco
Format: Artikel
Sprache:eng
Schlagworte:
6-minute walk distance
6-minute walk test
Annan medicin och hälsovetenskap
ataluren
Clinical trials
Codon, Nonsense - genetics
controlled trials
Demographics
Duchenne muscular dystrophy
efficacy
end-points
Health Care Sciences & Services
Humans
meta-analyses
Meta-analysis
multicenter
Muscular dystrophy
Muscular Dystrophy, Duchenne - drug therapy
Muscular Dystrophy, Duchenne - genetics
Mutation
nonsense mutation Duchenne muscular dystrophy
Other Medical and Health Sciences
Oxadiazoles - therapeutic use
Patients
randomized
randomized controlled trials
Randomized Controlled Trials as Topic
Statistical analysis
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Beschreibung
Zusammenfassung:Assess the totality of efficacy evidence for ataluren in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD). 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–
ISSN:2042-6305
2042-6313
DOI:10.2217/cer-2020-0095