Sarepta Therapeutics has announced the latest results from the 4053-101 study (see SKIP-NMD project for more details). This phase 1/2 clinical study is assessing the safety and efficacy of golodirsen, a drug that skips exon 53 in individuals with Duchenne muscular dystrophy. Muscle biopsies from the treated participants showed that the drug was able to skip exon 53. This had a positive effect on dystrophin production.
Please read Sarepta Therapeutics’ press release for more information.