Onasemnogene Abeparvovec
Zolgensma is a one-time gene therapy treatment for spinal muscular atrophy (SMA) that delivers a functional copy of the SMN gene to replace the defective or missing gene. It is approved for use in pediatric patients under 2 years of age with SMA, including those who are presymptomatic.
Zolgensma is a gene therapy that treats spinal muscular atrophy in young children by providing a functional SMN gene in a single intravenous infusion.
Key facts at a glance
- RxNorm
- 2170231
- Generic
- Zolgensma
- Class
- brand-name
- Brands
- —
- Availability
- rx
- Lactation
- unknown
- Routes
- —
- Forms
- —
- Regions
- US, EU, CN, JP
- Monitoring
- —
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Zolgensma is indicated for the treatment of spinal muscular atrophy (SMA) in patients under 2 years of age, including those with Type 1 SMA (the most severe form) and those who are presymptomatic but have been identified through newborn screening.
Zolgensma is an adeno-associated virus vector-based gene therapy that delivers a functional copy of the survival motor neuron (SMN) gene directly to motor neuron cells. The modified virus (AAV9) carries the SMN gene into cells, allowing them to produce the SMN protein necessary for motor neuron survival and function.
Zolgensma is administered as a single intravenous infusion and is intended for one-time use only. Serious potential side effects include acute liver injury, thrombocytopenia, and cardiac toxicity. Patients require regular monitoring of liver function and platelet counts. The treatment does not cross the blood-brain barrier, so it does not correct SMN deficiency in the central nervous system directly.
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 27, 2026. · Read our editorial policy