Current and emerging treatment strategies for Duchenne muscular dystrophy.

102 Among 174 boys with DMD due to confirmed premature stop codon mutations, a double-blind placebo-controlled study for 48 weeks showed a marginally significant improvement in 6MWT for those receiving low-dose (40 mg/kg/d) ataluren only; those on high-dose (80 mg/kg/d) treatment did not improve in their 6MWT when compared with placebo, and the results were attributed to ataluren’s bell-shaped dose–response curve. 103 Ataluren has received conditional approval by the European Medicines Agency since August 2014; 104 results from the confirmatory trial ( NCT02090959 ) is currently pending.

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