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.

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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.

Before and during treatment

Download Explaining SMA and ZOLGENSMA® (onasemnogene abeparvovec-xioi) to Others

Facts About SMA

The Facts About SMA provides you with easy-to-understand information about what causes SMA, signs and symptoms of SMA, and how quickly SMA can progress to a life-threatening condition. Learn more about SMA and bring this PDF to your doctor’s office to determine next steps.

Download the facts
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? What are the results of the clinical studies? How do you start treatment with ZOLGENSMA? Get answers to these questions and many more with this in-depth guide created just for caregivers.

Download the guide

After treatment

Download SMA After Treatment

SMA After Treatment

After your child has been treated with ZOLGENSMA, it is important to understand how to continue managing SMA and other health needs. This guide outlines potential members of a complete healthcare team, types of supportive therapies your child’s doctor may recommend, and how a nutritionist can help ensure nutritional needs for growth and development are being met.

Download the guide
Download the guide en Español
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 family’s experience with SMA and ZOLGENSMA. It covers facts about SMA, how ZOLGENSMA works, tips on how to help families living with SMA, and how they can help support the SMA community.

Download the handout

Prescribing Information

Download Prescribing Information

Full Prescribing Information provides comprehensive details about ZOLGENSMA, including dosage and administration, potential side effects, results from clinical trials, and much more.

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Download the Prescribing Information en Español

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 the patient experiences unexpected bleeding or bruising.
  • Thrombotic microangiopathy (TMA) has been reported to occur approximately one week after ZOLGENSMA infusion. Caregivers should seek immediate medical attention if the patient experiences any signs or symptoms of TMA, such as unexpected bruising or bleeding, seizures, or decreased urine output.

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 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 Novartis Gene Therapies, Inc. at 833-828-3947.

Please see the Full Prescribing Information.