# Eteplirsen (Exondys 51)

Source: https://ourhealthnetwork.com/drugs/eteplirsen
Data: FDA Orange Book and OpenFDA, refreshed periodically

## What is Eteplirsen?

Eteplirsen (brand name: Exondys 51) is an antisense oligonucleotide. It is prescribed to treat muscular dystrophy, duchenne.

## Brand names

Exondys 51

## Indications

Eteplirsen is prescribed for the treatment of muscular dystrophy and duchenne.

## Conditions treated

- [Muscular Dystrophy](https://ourhealthnetwork.com/conditions/muscular-dystrophy)
- [Duchenne](https://ourhealthnetwork.com/conditions/duchenne)

## Mechanism of action

Eteplirsen is designed to bind to exon 51 of dystrophin pre-mRNA, resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 51 skipping. Exon skipping is intended to allow for production of an internally truncated dystrophin protein, which was evaluated in Study 2 and Study 3 [ see Clinical Studies ( 14 ) ].

## Drug classification

- Established Pharmacologic Class: Antisense Oligonucleotide [EPC]
- Route of administration: INTRAVENOUS

## Common side effects

balance disorder and vomiting ( 6

## Sources and disclaimers

- FDA Orange Book and OpenFDA dataset
- This information is for educational purposes only and does not constitute medical advice. Always consult a healthcare professional before starting, stopping, or changing any medication.

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