Drisapersen, an antisense oligonucleotide that induces exon skipping in the dystrophin gene transcript, improves ambulation in patients with Duchenne muscular dystrophy (DMD), according to a phase II study. Investigators randomly assigned 53 patients with early-stage DMD to continuous or intermittent drisapersen, or to placebo. After 24 weeks of treatment, the patients who received continuous drisapersen, but not those in the other groups, showed a significant increase in 6-min walk distance.