About eteplirsen
Antisense oligonucleotide; exon-skipping therapy
Used for
- Treats Duchenne muscular dystrophy in patients with a confirmed exon 51 mutation.
Usual adult dose
30 mg/kg by intravenous infusion once weekly.
Usual dose in children
30 mg/kg by intravenous infusion once weekly; approved for eligible patients aged four years and older.
Common side effects
- Vomiting
- Balance problems
- Skin reactions near the infusion site
- Cough
Important warnings
- For exon 51 mutations only; genetic testing is required.
- Infusions can cause allergic reactions, including serious hypersensitivity.
- Kidney function may need monitoring; kidney injury occurred in animal studies.
- Administer by intravenous infusion under healthcare supervision.
Pregnancy
There are no adequate studies in pregnant people; potential risks are unknown.
Breastfeeding
It is unknown whether eteplirsen passes into breast milk; effects on infants are unknown.
AI-generated from general medical knowledge, not from this product's official leaflet. It may be incomplete or out of date. Not medical advice — ask a pharmacist or doctor before making any decision.