To date, this intense activity has only involved Duchenne muscular dystrophy, with the main therapeutic options being as follows: (i) reframing the DMD transcript via exon skipping which is induced by a synthetic antisense oligoribonucleotide (eteplirsen, golodirsen, casimersen and drisapersen) omitting exon 51, 53, 45 or 44 respectively; (ii) ribosomal read-through of nonsense variants (ataluren), which is based on the known low specificity of tRNAs in cognate codon recognition; (iii) gene therapy based on minigenes which encode shortened forms of dystrophin. The gene discussed is DMD; the disease is Duchenne muscular dystrophy.