eteplirsen

Eteplirsen is an antisense oligonucleotide designed to treat Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping. Developed by Sarepta Therapeutics, it works by binding to exon 51 of the dystrophin pre-mRNA, causing this exon to be excluded during splicing. This "skipping" allows for the production of an internally truncated but functional dystrophin protein, potentially slowing the progression of muscle weakness and wasting. It was the first drug approved by the FDA for DMD, receiving accelerated approval based on the surrogate endpoint of increased dystrophin expression in skeletal muscle.

Brand names

Exondys 51Exondys51Exondys-51

Other names

eteplirsenum