Eravacycline
Elevidys is a gene therapy designed to treat Duchenne muscular dystrophy in patients with a confirmed mutation in the dystrophin gene who are amenable to this treatment. It works by delivering a functional copy of the gene that codes for a shortened form of dystrophin (micro-dystrophin) to muscle cells. This medication is administered as a single intravenous infusion and is specifically indicated for patients aged 4 through 5 years with Duchenne muscular dystrophy.
Elevidys is a one-time gene therapy infusion for young children with Duchenne muscular dystrophy that delivers a functional dystrophin gene to slow disease progression.
Key facts at a glance
- RxNorm
- 2641681
- Generic
- Elevidys
- 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.
Elevidys is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients with a confirmed DMD gene mutation who are amenable to treatment with this specific therapy. It is currently approved for use in children aged 4 through 5 years.
Elevidys is a recombinant gene therapy that uses a modified adeno-associated virus (AAV) vector to deliver a transgene encoding a functional micro-dystrophin protein directly to muscle tissue. This micro-dystrophin is a shortened but functional version of the dystrophin protein that is missing or defective in patients with DMD.
Elevidys is administered as a single intravenous infusion. Potential serious side effects include immune-mediated myositis and acute liver injury. Patients must be monitored closely before and after treatment. As this is a one-time treatment, long-term data on durability of response continues to be collected. Genetic testing to confirm mutation eligibility is required before treatment.
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 July 26, 2026. · Read our editorial policy