Delandistrogene Moxeparvovec-Rokl
Delandistrogene moxeparvovec-rokl is a gene therapy medication designed to treat certain forms of muscular dystrophy. It works by delivering a functional copy of the dystrophin gene to muscle cells using a viral vector, helping to restore dystrophin protein production in patients with specific genetic mutations.
A gene therapy that delivers the dystrophin gene to muscle cells to treat certain forms of muscular dystrophy.
Key facts at a glance
- RxNorm
- 2641674
- Generic
- Delandistrogene Moxeparvovec-Rokl
- Class
- pin-variant
- Brands
- Elevidys
- Availability
- rx
- Lactation
- unknown
- Routes
- —
- Forms
- —
- Regions
- US, EU, CN, JP
- Monitoring
- —
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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.
Treatment of muscular dystrophy in patients with specific genetic mutations amenable to this gene therapy approach.
Delivers a functional dystrophin gene to muscle cells via a recombinant viral vector, allowing cells to produce a truncated but functional dystrophin protein that helps protect and stabilize muscle fibers.
Administered as a single intravenous infusion. Requires genetic testing to confirm eligible mutations. Pre-existing immunity to the viral vector may affect eligibility. Potential immune responses and liver enzyme elevations should be monitored. Patients should be evaluated for adequate vector-neutralizing antibody levels 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 August 25, 2026. · Read our editorial policy