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A picture of Matteo, a child with SMA, drawing with chalk on a driveway

 
Choose the only SMA gene replacement therapy    
 

Matteo has 2 copies of SMN2 gene 
Treated at ~1 month old

ZOLGENSMA® (onasemnogene abeparvovec-xioi) stops the progression of spinal muscular atrophy (SMA) by helping children meet milestones, like sitting, standing, walking on their own, and more.*
ZOLGENSMA is a prescription gene replacement therapy for the treatment of SMA in children less than 2 years old.

*Results and outcomes vary among children based on several factors, including how far their SMA symptoms progressed prior to receiving treatment.

ZOLGENSMA—Over 9 years of follow-up data for children with SMA

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Want to know more about ZOLGENSMA?

Learn how ZOLGENSMA targets the root cause of SMA with a one-time dose
 

How ZOLGENSMA works
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Curious about the ZOLGENSMA treatment experience?

Early treatment is important. Learn how to get started with ZOLGENSMA

The path to ZOLGENSMA
Aniya, a child with SMA, looking forward as her parents, Hailey and Will, gaze at her with pride

Aniya has 4 copies of SMN2.

See how her family pushed for early treatment and how that made a big difference

See family stories
4,000+ children with SMA have been treated with ZOLGENSMA as of August 2024. *Globally, including clinical trials, commercially, and through the managed access programs.
Malachi, a child with SMA, in a wheelchair staring off into the distance

Novartis Patient Support™ 

Guidance for families from the moment they choose ZOLGENSMA—including understanding insurance and life after treatment—answering questions along the way.

Learn about Novartis Patient Support