Sarepta Therapeutics has released positive preliminary results from one of its clinical trials testing exon skipping in Duchenne muscular dystrophy. The study drug, casimersen, targets exon 45 of the dystrophin gene. The investigators have found increased levels of dystrophin in muscles of boys treated with the drug for 11 months. Information on the safety and efficacy of the treatment will only be available at a later stage. However, based on these initial results, Sarepta hopes to request approval from the US Food and Drug Administration in the middle of 2019.
For more information, read Sarepta’s press release.