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The path to treatment with ZOLGENSMA

Evelyn has 2 copies of SMN2  gene
Treated at ~2 months old

The path to treatment with ZOLGENSMA

Take these next steps for your child

Early diagnosis and early treatment can help stop the progression of spinal muscular astrophy (SMA).* When that treatment is ZOLGENSMA® (onasemnogene abeparvovec-xioi), we’ll be there with you through every step of your treatment journey to help ensure the process goes smoothly. Here’s what you can expect:

Important lab tests

Your child’s doctor will schedule lab tests to help determine eligibility for your child. It’s generally best to have the tests done as soon as you can so that your child can get treated as quickly as possible; work with your doctor to schedule them.

Your child’s doctor submits prescription and consent forms

While waiting for the results of your child’s lab tests, ask their doctor to submit two forms: a prescription form and a consent form to enroll you in the OneGene Program®, which provides additional support before, during, and after treatment.

Working with your insurance

Your OneGene Program team can help with the verifications and authorizations required for treatment.

Preparing for treatment day

To make the day go smoothly, confirm the logistics, ensure all blood work has been completed, watch for signs of infection, and start the prescribed corticosteroid. And, schedule follow-up appointments with your child’s doctor.

Treatment Day!

Administer a second corticosteroid dose, and confirm follow-up appointments. Expect the infusion to take about 60 minutes. You will be asked to remain at the treatment center for a period of time after treatment. Ask your doctor in advance how long you’ll be required to stay.

Moving forward after SMA treatment

Immediately after infusion

You and your child’s doctor will schedule a number of follow-up appointments over the next 3 months or longer for follow-up lab work.

After-treatment care plan

Your Care Plan After Treatment

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Looking to the future

Your OneGene Program team will reach out to follow up. To help meet your long-term goals, speak with your child’s neuromuscular specialist, and maintain check-ups.

If you still have questions about what to expect next, please reach out to your doctor.

*Results and outcomes vary among children based on several factors, including how far their SMA symptoms progressed prior to receiving treatment.

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Steps to Treating With ZOLGENSMA brochure

Want to know more? Download our Step-by-Step Guide to treatment with ZOLGENSMA

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Learn about other families’ experiences with ZOLGENSMA

ZOLGENSMA stories

Important Safety Information

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

  • ZOLGENSMA can increase liver enzyme levels and cause acute serious liver injury or acute liver failure which could result in death.
  • 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, if the patient misses a dose of corticosteroid or vomits it up, or if the patient experiences a decrease in alertness.

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

  • Infections before or after ZOLGENSMA infusion can lead to more serious complications. Caregivers and close contacts with the patient should follow infection prevention procedures. Contact the patient's doctor immediately if the patient experiences any signs of a possible 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 generally occur within the first two weeks after ZOLGENSMA infusion. 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.
  • There is a theoretical risk of tumor development with gene therapies such as ZOLGENSMA. Contact the patient’s doctor and Novartis Gene Therapies, Inc. (1-833-828-3947) if a tumor develops.

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 influenza and respiratory syncytial virus (RSV) is recommended and vaccination status should be up-to-date prior to ZOLGENSMA administration. Please consult the patient's doctor.

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 patient body waste for one 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 1-833-828-3947.

Please see the FullPrescribing Information.

Tap or scroll down to read full Important Safety Information and IndicationArrow icon

Important Safety Information

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

  • ZOLGENSMA can increase liver enzyme levels and cause acute serious liver injury or acute liver failure which could result in death.
  • 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, if the patient misses a dose of corticosteroid or vomits it up, or if the patient experiences a decrease in alertness.

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

  • Infections before or after ZOLGENSMA infusion can lead to more serious complications. Caregivers and close contacts with the patient should follow infection prevention procedures. Contact the patient’s doctor immediately if the patient experiences any signs of a possible 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 generally occur within the first two weeks after ZOLGENSMA infusion. 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.
  • There is a theoretical risk of tumor development with gene therapies such as ZOLGENSMA. Contact the patient’s doctor and Novartis Gene Therapies, Inc. (1-833-828-3947) if a tumor develops.

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 influenza and respiratory syncytial virus (RSV) is recommended and vaccination status should be up-to-date prior to ZOLGENSMA administration. Please consult the patient’s doctor.

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 patient body waste for one 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 1-833-828-3947.

Please see the Full Prescribing Information.

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The information provided in this site is intended only for audiences of the United States. This information does not take the place of talking to your health care professional about medical conditions and treatments. If you have questions about ZOLGENSMA after reading this information, ask your health care professional.

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