Eteplirsen
Vyondys 53 (golodirsen) is a prescription medication used to treat Duchenne muscular dystrophy (DMD) in patients with a confirmed genetic mutation amenable to exon 53 skipping. It is an antisense oligonucleotide that helps produce a shorter, partially functional form of the dystrophin protein, which is essential for muscle function and is deficient in patients with DMD.
Vyondys 53 is a targeted therapy for certain patients with Duchenne muscular dystrophy that helps restore a partial form of the dystrophin protein.
Key facts at a glance
- RxNorm
- 2267212
- Generic
- Vyondys 53
- 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.
Vyondys 53 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 covers approximately 8-10% of all DMD cases. It is approved for use in patients of all ages who meet this genetic criterion.
Vyondys 53 is an antisense oligonucleotide that binds specifically to exon 53 of the dystrophin pre-mRNA. This binding causes the cellular machinery to skip over exon 53 during mRNA processing, resulting in the production of a truncated but partially functional dystrophin protein. This shortened dystrophin can provide some protective benefits to muscle fibers, potentially slowing disease progression.
Vyondys 53 is administered by intravenous (IV) infusion every week. Common side effects may include headache, fever, and abdominal pain. Serious risks include potential kidney toxicity, requiring regular monitoring of kidney function during treatment. It may also cause infusion-related reactions. This medication is specifically for patients with DMD mutations amenable to exon 53 skipping and is not appropriate for other forms of muscular dystrophy. Treatment should be initiated and monitored by physicians experienced in managing DMD.
Official drug label
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This information is for educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment.
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