In a phase 2b trial that included 174 patients, ataluren, designed to permit stop codon read-through, was associated with a marginally significant improvement in the 6MWT compared with the placebo [ 9 ].
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In a phase 2b trial that included 174 patients, ataluren, designed to permit stop codon read-through, was associated with a marginally significant improvement in the 6MWT compared with the placebo [ 9 ].