Your guide to ZOLGENSMA

A Caregiver’s Guide to ZOLGENSMA is specifically designed to help caregivers learn more about spinal muscular atrophy (SMA) and ZOLGENSMA® (onasemnogene abeparvovec-xioi). It covers everything from what causes SMA to how ZOLGENSMA works in the body and the necessary steps to start treatment. You will also learn about other families and their experience with ZOLGENSMA and how they’re doing after treatment.

Download the guide
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Your guide to ZOLGENSMA

A Caregiver’s Guide to ZOLGENSMA is specifically designed to help caregivers learn more about spinal muscular atrophy (SMA) and ZOLGENSMA® (onasemnogene abeparvovec-xioi). It covers everything from what causes SMA to how ZOLGENSMA works in the body and the necessary steps to start treatment. You will also learn about other families and their experience with ZOLGENSMA and how they’re doing after treatment.

Download the guide

Overview of the guide

Learn more about each section and what to expect at every step toward treatment.

A Caregiver's Guide to ZOLGENSMA® (onasemnogene abeparvovec-xioi) - What is SMA?

What is SMA?

This section of the guide helps you understand what causes SMA and why it’s critical to talk to your child’s doctor immediately if you see any of the signs and symptoms of SMA. It also explains the range of severity in people affected by SMA who haven’t received treatment and how quickly it may lead to the need for breathing and eating support.


A Caregiver's Guide to ZOLGENSMA® (onasemnogene abeparvovec-xioi) - How ZOLGENSMA works

How ZOLGENSMA works

This section explains how ZOLGENSMA treats SMA with a one-time-only dose. You will learn how the new gene reaches its destination, how it replaces the function of the missing or nonworking gene, and what the new gene does to start helping the body function properly.


A Caregiver's Guide to ZOLGENSMA® (onasemnogene abeparvovec-xioi) - Results from the ZOLGENSMA clinical studies

Results from the ZOLGENSMA clinical studies

ZOLGENSMA was evaluated in 2 open-label clinical studies. See the results of both studies within the brochure.


A Caregiver's Guide to ZOLGENSMA® (onasemnogene abeparvovec-xioi) - Steps to starting ZOLGENSMA

See the steps to starting ZOLGENSMA

Once you and your doctor have chosen ZOLGENSMA, it’s important to take the necessary steps to start treatment and learn about the resources available to your family. This section covers the types of tests needed before the infusion and how the OneGene Program offers guidance and support at every step of the treatment journey.

Learn more about SMA and ZOLGENSMA

Download A Caregiver’s Guide to ZOLGENSMA

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.