EXONDYS 51 (eteplirsen) is an antisense oligonucleotide approved under accelerated approval for treating Duchenne muscular dystrophy patients with confirmed DMD gene mutations amenable to exon 51 skipping, based on its ability to increase dystrophin protein levels in skeletal muscle, though confirmatory trials for clinical benefit verification are still pending.

Therapeutic Mechanism and Efficacy of Eteplirsen in DMD Management

EXONDYS 51 represents a targeted molecular approach for Duchenne muscular dystrophy (DMD) treatment. As a phosphorodiamidate morpholino oligomer, eteplirsen binds specifically to exon 51 of dystrophin pre-mRNA, facilitating exon skipping during mRNA processing. This mechanism enables production of an internally truncated but partially functional dystrophin protein in patients with genetic mutations amenable to exon 51 skipping.