European Medicines Agency review of ataluren for the treatment of ambulant patients aged 5 years and older with Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene

MeSH terms

• Adolescent
• Adult
• Child
• Child, Preschool
• Clinical Trials, Phase II as Topic
• Codon, Nonsense*
• Drug Approval*
• Dystrophin / genetics*
• Humans
• Male
• Muscular Dystrophy, Duchenne / drug therapy*
• Muscular Dystrophy, Duchenne / genetics*
• Oxadiazoles / therapeutic use*
• Randomized Controlled Trials as Topic
• Treatment Outcome
• Young Adult