Eteplirsen

EXONDYS 51 is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping.

Prescribing label

Selected manufacturer label information, organised into consistent sections by Sofidex. This source is written for healthcare professionals; ask your pharmacist to explain instructions that are unclear. Read the full label at DailyMed ↗

Drug facts

Used for: This indication is approved under accelerated approval based on an increase in dystrophin in skeletal muscle observed in some patients treated with EXONDYS 51.
Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials.

What it is used for

EXONDYS 51 is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping. This indication is approved under accelerated approval based on an increase in dystrophin in skeletal muscle observed in some patients treated with EXONDYS 51. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials.

Who should not take it

None.

Storing it

Store EXONDYS 51 at 2°C to 8°C (36°F to 46°F). Do not freeze. Protect from light and store EXONDYS 51 in the original carton until ready for use.

Cite this page

NLM: sofidex [Internet]. Daloreum; c2026. Eteplirsen; [reviewed 2026 Aug 2; cited 2026 Nov 10]. Available from: https://sofidex.com/patients/medicines/eteplirsen
APA 7th: sofidex. (2026, August 2). Eteplirsen. Daloreum. Retrieved November 10, 2026, from https://sofidex.com/patients/medicines/eteplirsen
AMA 11th: Eteplirsen. sofidex. Reviewed August 2, 2026. Accessed November 10, 2026. https://sofidex.com/patients/medicines/eteplirsen

The cited date is today’s, because a citation records when you read the page. The reviewed date is when a clinician last checked it.