New drug 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, USA, 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.

"I've been doing this for more than 40 years and this is one of the most exciting developments we've seen," says Jerry Mendell, lead author of the study and director of the Center for Gene Therapy at Nationwide Children's, adding: "It offers great hope to patients with Duchenne muscular dystrophy and their families."