Trial finds eteplirsen improves walking performance for Duchenne muscular dystrophy patients

Results from a clinical trial of eteplirsen, a drug designed to treat Duchenne muscular dystrophy, suggest that the therapy allows participants to walk farther than people treated with placebo and dramatically increases production of a protein vital to muscle growth and health. The study, led by a team in The Research Institute at Nationwide Children's Hospital, is the first of its kind to show these results from an exon-skipping drug - a class of therapeutics that allows cells to skip over missing parts of the gene and produce protein naturally...

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