One of the most common types of mutations in the dystrophin gene occurs when a piece of the code in the middle of the gene is missing or deleted. By skipping additional segments of the dystrophin code called exons, the deletion can shift from an out-of-frame deletion to an in-frame deletion. Typically an in-frame deletion results in a smaller, but still functional, dystrophin protein. This shortened protein is expected to act in a similar way to normal dystrophin, and so relieve some symptoms of Duchenne and hopefully result in a more mild presentation. EXONDY 51® is for use in patients with a mutation in the dystrophin gene that is amenable to exon 51 skipping.

Status & Important Information

• EXONDYS 51 (eteplirsen) is for individuals with Duchenne muscular dystrophy who have a specific genetic variant amenable to exon 51 skipping.
• EXONDYS 51 is a once-weekly infusion, usually through an IV (intravenous) or a port. 
• It helps the body make a shorter but functional dystrophin protein, which aims to slow down muscle damage and progression.

For additional resources regarding eligibility and access, view PPMD’s Insurance Access & Coverage Roadmap.

Sponsor

This program is sponsored by Sarepta Therapeutics.

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