Viltolarsen
Viltepso (viltolarsen) is a prescription medication used to treat Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping. It is an antisense oligonucleotide that works by helping the body produce a shorter form of dystrophin protein, which may help slow disease progression.
Viltepso is a gene-targeted therapy for Duchenne muscular dystrophy that helps produce a functional dystrophin protein in patients with eligible genetic mutations.
Key facts at a glance
- RxNorm
- 2389845
- Generic
- Viltepso
- Class
- brand-name
- Brands
- —
- Availability
- rx
- Lactation
- unknown
- Routes
- —
- Forms
- —
- Regions
- US, EU, CN, JP
- Monitoring
- —
This page is for informational purposes only.
Do not use this information to diagnose yourself or to replace a physician's advice. Always consult a qualified healthcare provider before starting, stopping, or changing any medication. If you believe you are experiencing a medical emergency, call your local emergency number immediately.
Viltepso is indicated for the treatment of Duchenne muscular dystrophy in patients with a confirmed mutation of the DMD gene that is amenable to exon 53 skipping. This genetic mutation accounts for approximately 8% of all DMD cases.
Viltepso is an antisense oligonucleotide that binds to a specific region of the DMD gene pre-mRNA, causing the body to skip over (exclude) exon 53 during processing. This enables the production of a shortened but partially functional dystrophin protein, which helps protect muscle cells from damage.
Viltepso is administered via intravenous infusion once weekly. Common side effects may include upper respiratory tract infections, injection reactions, and cough. Regular monitoring of kidney function is recommended during treatment. Patients should be monitored for hypersensitivity reactions during infusion.
Official drug label
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This content is for general informational and educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment. Always seek the guidance of a qualified healthcare provider with any questions you have about a medical condition, and never disregard professional medical advice or delay seeking it because of something you read here.
If you think you may have a medical emergency, call your local emergency number or go to the nearest emergency department immediately.
Medically reviewed by WellAlly Editorial Team Last reviewed August 26, 2026. · Read our editorial policy