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. 2021 Jul 9;4(3):232–240. doi: 10.31662/jmaj.2021-0019

Table 1.

Antisense Oligonucleotides (ASOs) to Treat Duchenne Muscular Dystrophy (DMD).

Name ASO Target exon Phase
Nucleic acid Size Route
Eteplirsen PMO 25 IV 51 Approved
Golodirsen PMO 25 IV 53 Approved
Viltolarsen PMO 21 IV 53 Approved
Casimersen PMO 25 IV 45 Approval stage
Renadirsen ENA/2'RNA 18 SC 45 I/II

PMO: phosphorodiamidate morpholino; ENA: 2'-O,4'-C-ethylene-bridged nucleic acid; 2'RNA: 2'-O-methyl RNA; IV: intravenous injection; SC: subcutaneous injection