ZOLGENSMA® (onasemnogene abeparvovec-xioi) is a prescription gene therapy used to treat children less than 2 years old with spinal muscular atrophy (SMA). It targets the genetic root cause of SMA with a one-time-only dose and replaces the function of the missing or nonworking survival motor neuron 1 (SMN1) gene with a new, working copy of a human SMN gene.
Watch how it worksCatch up with some of the children with SMA Type 1 who took part in group 2 of the START clinical study of ZOLGENSMA. Hear about why they chose ZOLGENSMA and see how they are doing years after treatment.
Watch their videosWhat causes SMA? How does ZOLGENSMA work in the body? How do you start treatment with ZOLGENSMA? Get these answers and many more in this in-depth guide created just for caregivers.
Get the guideLearn about the clinical study results in children diagnosed with SMA.
View ZOLGENSMA resultsAfter receiving a disease-modifying treatment like ZOLGENSMA, the SMA journey continues and SMA Atlas is here to help. SMA Atlas is where you will find an abundance of helpful resources about living with SMA, tips from experienced caregivers on the importance of building a complete healthcare team, and much more.
Embark on the next chapter with SMA AtlasThe OneGene Program™ is a team of highly trained and dedicated people who will support and guide you and your family before, during, and after treatment.
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What is the most important information I should know about
ZOLGENSMA?
What is the most important information I should know about
ZOLGENSMA?
What should I watch for before and after infusion with
ZOLGENSMA?
What do I need to know about vaccinations and ZOLGENSMA?
Do I need to take precautions with the patient’s bodily waste?
What are the possible or likely side effects of ZOLGENSMA?
What is ZOLGENSMA?
ZOLGENSMA is a prescription gene therapy used to treat children less than 2 years
old with spinal muscular atrophy (SMA). ZOLGENSMA is given as a one-time infusion
into a vein. ZOLGENSMA was not evaluated in patients with advanced SMA.
The safety information provided here is not comprehensive. Talk to the patient’s doctor about any side effects that bother the patient or that don’t go away.
You are encouraged to report suspected side effects by contacting the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch, or AveXis at 833-828-3947.
Please see the Full Prescribing Information.