(From the September 2013 Issue of MedStat)
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. Jerry Mendell, MD, director of the Center for Gene Therapy at Nationwide Children’s, is lead author of the study which appears in the journal Annals of Neurology.
Read the news release.