I’ll always remember the day

we received the one-time-only dose for SMA

See how ZOLGENSMA works

ZOLGENSMA targets the genetic root cause of SMA

ZOLGENSMA (pronounced zol-JEN-smuh) replaces the function of the missing or nonworking survival motor neuron 1 (SMN1) gene with a new, working copy of a human SMN gene that helps motor neuron cells work properly and survive.

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Evelyn, who has SMA Type 1, by a window Play Button
Malachi, who has SMA Type 1, laughing while he holds a dandelion Play Button
Donovan, who has SMA Type 1, holding a blue toy Play Button

Meet children from the study

Twelve children with SMA Type 1 took part in group 2 of the START clinical study of ZOLGENSMA.

Watch some of the families share their SMA journey, hear about the day their child was given ZOLGENSMA, and learn how their child is doing after treatment.

Watch their videos
Evelyn, who has SMA Type 1, by a window Play Button
Malachi, who has SMA Type 1, laughing while he holds a dandelion Play Button
Donovan, who has SMA Type 1, holding a blue toy Play Button

The OneGene Program

The 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.

Meet your support team
Remember the day - ZOLGENSMA® (onasemnogene abeparvovec-xioi) calendar

A one-time-only dose

ZOLGENSMA is changing how children with SMA can be medically treated with one dose given intravenously (IV) over the course of 60 minutes.

Learn about ZOLGENSMA
Download A Caregiver’s Guide to ZOLGENSMA® (onasemnogene abeparvovec-xioi)

A Caregiver’s Guide to ZOLGENSMA

What causes SMA? How does ZOLGENSMA work in the body? How do you start ZOLGENSMA treatment? Get these answers and many more in this in-depth guide created just for caregivers.

Get the guide

Adalyne, treated at ~2½ months and pictured at 3 years, has SMA Type 1.

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Getting started on ZOLGENSMA

Know what to expect before, during, and after treatment.

Start now
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Results that make a difference

Learn about the results of 2 clinical studies.

View study results
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Our experiences with ZOLGENSMA

Watch families talk about the day their child was given ZOLGENSMA and how their child is doing after treatment.

Watch the videos

Important Safety Information

What is the most important information I should know about ZOLGENSMA?

  • ZOLGENSMA can cause acute serious liver injury. Liver enzymes could become elevated and may reflect acute serious liver injury in children who receive ZOLGENSMA.
  • Patients will receive an oral corticosteroid before and after infusion with ZOLGENSMA and will undergo regular blood tests to monitor liver function.

Important Safety Information

What is the most important information I should know about ZOLGENSMA?

  • ZOLGENSMA can cause acute serious liver injury. Liver enzymes could become elevated and may reflect acute serious liver injury in children who receive ZOLGENSMA.
  • Patients will receive an oral corticosteroid before and after infusion with ZOLGENSMA and will undergo regular blood tests to monitor liver function.
  • Contact the patient’s doctor immediately if the patient’s skin and/or whites of the eyes appear yellowish, or if the patient misses a dose of the corticosteroid or vomits it up.

What should I watch for before and after infusion with ZOLGENSMA?

  • Viral respiratory infections before or after ZOLGENSMA infusion can lead to more serious complications. Contact the patient’s doctor immediately if you see signs of a possible viral respiratory infection such as coughing, wheezing, sneezing, runny nose, sore throat, or fever.
  • Decreased platelet counts could occur following infusion with ZOLGENSMA. Seek immediate medical attention if a patient experiences unexpected bleeding or bruising.

What do I need to know about vaccinations and ZOLGENSMA?

  • Talk with the patient’s doctor to decide if adjustments to the vaccination schedule are needed to accommodate treatment with a corticosteroid.
  • Protection against respiratory syncytial virus (RSV) is recommended.

Do I need to take precautions with the patient’s bodily waste?

Temporarily, small amounts of ZOLGENSMA may be found in the patient’s stool. Use good hand hygiene when coming into direct contact with bodily waste for 1 month after infusion with ZOLGENSMA. Disposable diapers should be sealed in disposable trash bags and thrown out with regular trash.

What are the possible or likely side effects of ZOLGENSMA?

The most common side effects that occurred in patients treated with ZOLGENSMA were elevated liver enzymes and vomiting.

Indication

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 the 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.