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Frequently asked questions

Eli has 2 copies of SMN2 gene
Treated at ~4 months old

Frequently asked questions

Want to learn more?

Below are some common questions about ZOLGENSMA®  (onasemnogene abeparvovec-xioi) and the support services available. For medical questions, your doctor should always be your primary resource for information.

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Can I wait to treat SMA?

The sooner treatment with ZOLGENSMA is given, the sooner muscle function may be preserved. For children with 2, 3, or 4 SMN2 backup gene copies, early treatment with ZOLGENSMA is recommended to halt spinal muscular atrophy (SMA) progression.

What is ZOLGENSMA?

ZOLGENSMA is a gene therapy approved by the US Food and Drug Administration (FDA) for children less than 2 years old with spinal muscular atrophy (SMA). ZOLGENSMA is:

  • A one-time-only dose
  • Given intravenously (IV)
  • An infusion that takes 60 minutes

How is ZOLGENSMA given?

ZOLGENSMA is given intravenously as a one-time infusion. The actual infusion takes about 60 minutes. Learn more about the step-by-step process of receiving ZOLGENSMA.

What if my child’s adeno-associated virus 9 (AAV9) antibody levels are too high to receive ZOLGENSMA?

Talk to your doctor about monitoring this antibody level. If your child’s immune system has built up a certain level of anti-AAV9 antibodies, they might not be eligible to receive ZOLGENSMA right away.

Ask your child’s doctor to continue monitoring your child’s AAV9 antibody levels—they can decrease over time and your child may become eligible to receive ZOLGENSMA.

How will my child’s doctor follow up after a ZOLGENSMA infusion?

You and your child’s doctor will schedule a number of follow-up appointments over the next 3 months or longer. Your child’s doctor will be carefully looking at liver function, as well as other blood work and clinical exams.

Your child’s monitoring schedule over 3 months:

All results are compared against a baseline assessment taken before your child’s infusion recording liver function, creatinine, complete blood count (including hemoglobin and platelet count), and troponin-I.

Can I get help with insurance?

Through our OneGene Program®, families are assigned a Case Coordinator who acts as a go-between to connect you to resources you may need and assists doctors with insurance processes. Other support includes verification of insurance benefits and coordination of financial assistance programs for eligible children.

Does ZOLGENSMA halt SMA progression?

Early treatment is recommended for all children with SMA. Results and outcomes vary among children based on several factors, including how far their SMA symptoms progressed prior to receiving treatment. Please read the results of the 3 trials carefully  to learn about the potential risks and potential benefits of ZOLGENSMA.

How does gene therapy work?

ZOLGENSMA is designed to target the genetic root cause of SMA with a one-time dose by replacing the function of the missing or nonworking survival motor neuron 1 (SMN1) gene. The new gene tells motor neuron cells to produce more survival motor neuron (SMN) protein. Motor neuron cells need SMN protein to survive and support muscle functions. Read more about how ZOLGENSMA works.

What are the possible or likely side effects of ZOLGENSMA?

The most common side effects that occurred in children treated with ZOLGENSMA were elevated liver enzymes and vomiting. 
Read about ZOLGENSMA safety information.

How long do the benefits from ZOLGENSMA last?

Gene therapy in SMA is designed to keep producing SMN protein in a child’s body, helping to preserve muscle function. 
Please read the results of the 3 trials carefully to learn about the risks and benefits of ZOLGENSMA. Results and outcomes vary among children based on several factors, including how far their SMA symptoms progressed prior to receiving treatment.

Get your child started with ZOLGENSMA

See the path to ZOLGENSMA

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 healthcare professional about medical conditions and treatments. If you have questions about ZOLGENSMA after reading this information, ask your healthcare professional.

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