Eteplirsen

Eteplirsen (brand name Exondys 51) is an antisense oligonucleotide therapy designed to induce exon 51 skipping in the dystrophin gene, enabling partial restoration of dystrophin protein expression in patients with Duchenne muscular dystrophy carrying specific exon 51 mutations, Eteplirsen is extensively applied in genetic disease research, RNA-targeted therapy development, and translational studies of muscular dystrophy.