Notably, exon 51 is frequently adjacent to frame-disrupting deletions in DMD patients and has been targeted in clinical trials for oligonucleotide-based exon skipping with promising early therapeutic results.19,26,27 An ongoing clinical trial for the exon 51 skipping compound eteplirsen recently reported a significant functional benefit across 48 weeks, with an average of 47% dystrophin-positive fibers compared with baseline. The gene discussed is DMD; the disease is Duchenne muscular dystrophy.