Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial

Medicine & Life Sciences

• ataluren 100%
• Duchenne Muscular Dystrophy 63%
• Nonsense Codon 62%
• Placebos 37%
• Dystrophin 16%
• Least-Squares Analysis 15%
• Occupational Groups 14%
• Random Allocation 13%
• Intention 12%
• Walk Test 8%
• Latin America 8%
• Intention to Treat Analysis 7%
• North America 7%
• Therapeutics 6%
• Caregivers 5%
• Adrenal Cortex Hormones 5%
• Disease Progression 5%
• Reference Values 5%
• Population 5%
• Parents 5%
• Databases 4%
• Safety 3%
• Mutation 3%
• Liver 3%
• Proteins 2%