Exondys 51

Exondys 51 (eteplirsen) is a medication designed to treat certain forms of Duchenne muscular dystrophy (DMD), a genetic disorder that causes progressive muscle weakness. It works by helping the body produce a functional version of dystrophin, a protein essential for healthy muscle function that is missing or defective in DMD. Specifically, Exondys 51 targets a genetic mutation affecting exon 51 of the dystrophin gene, encouraging the body to skip this faulty segment and produce more functional protein. While it doesn't cure DMD, it may slow muscle deterioration and improve quality of life for some patients.