title: A Phase I/IIa Clinical Trial in Duchenne Muscular Dystrophy Using Systemically Delivered Morpholino Antisense Oligomer to Skip Exon 53 (SKIP-NMD).
mesh_terms: D016189:Dystrophin / Q000235:genetics; D005091:Exons; D006801:Humans; D008297:Male; D060172:Morpholinos / Q000627:therapeutic use*; D020388:Muscular Dystrophy, Duchenne / Q000235:genetics / Q000628:therapy*
chemical_list: D016189:Dystrophin; D060172:Morpholinos