Eteplirsen

Eteplirsen is a medication designed to treat Duchenne muscular dystrophy (DMD), a genetic disorder that weakens muscles over time. It works by helping the body produce a functional form of a protein called dystrophin, which is missing or defective in people with DMD. By using a technique called exon skipping, etiplirsen encourages cells to produce more dystrophin, potentially slowing muscle degeneration. It is administered through regular intravenous infusions and is typically prescribed for patients with specific genetic mutations affecting exon 51 of the dystrophin gene.