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.
References
Voit, T. et al. Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study. Lancet Neurol. 10.1016/S1474-4422(14)70195-4
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Exon-skipping approach shows potential in patients with Duchenne muscular dystrophy. Nat Rev Neurol 10, 547 (2014). https://doi.org/10.1038/nrneurol.2014.171
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DOI: https://doi.org/10.1038/nrneurol.2014.171