Helpful ZOLGENSMA downloads

Whether you are just exploring your treatment options or have finished treatment with ZOLGENSMA® (onasemnogene abeparvovec-xioi), the resources below can help wherever your family is in their spinal muscular atrophy (SMA) journey.

Helpful ZOLGENSMA downloads

Whether you are just exploring your treatment options or have finished treatment with ZOLGENSMA® (onasemnogene abeparvovec-xioi), the resources below can help wherever your family is in their spinal muscular atrophy (SMA) journey.

Download Steps to Treatment

Steps to Treatment

Learn about the steps to take toward starting ZOLGENSMA and the dedicated support that is available during the treatment journey and beyond.

Download the steps
Download a Caregiver's Guide to ZOLGENSMA

A Caregiver’s Guide to ZOLGENSMA

This helpful guide covers everything from facts about SMA and how ZOLGENSMA works to results from the clinical studies and steps to starting treatment.

Download the guide
Download SMA After Treatment

SMA After Treatment

Know what to expect and how to prepare for life with SMA after treatment with ZOLGENSMA. Learn about additional therapies, accommodations, and support that may be needed.

Download the guide
Download Explaining SMA and ZOLGENSMA to Others

Explaining SMA and ZOLGENSMA to Others

Share this handout to help people who have questions and want to know more about your child’s and family’s experience with SMA and ZOLGENSMA.

Download the handout
Download Facts About SMA

Facts About SMA

Get the Facts About SMA to bring to your child’s doctor to determine next steps.

Download the facts

Important Safety Information

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

  • Liver enzymes could become elevated and cause 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?

  • Liver enzymes could become elevated and cause 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.