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Two parents with their toddler and baby girls in a park-like area

Connecting with the SMA community

Ellie has 2 copies of SMN2  gene
Treated at 15 days old

Receiving a spinal muscular atrophy (SMA) diagnosis can be overwhelming and confusing. Just know you’re not alone. In addition to your Novartis Patient Support™ Care Coordinators, there are also a number of groups and events that can provide you with additional information and connect you with a community of other families and caregivers who share your experience.

Cure SMA logo

Cure SMA

Cure SMA is an organization dedicated to helping those in the SMA community. From practical support programs to funding SMA research, they help caregivers and patients access care, including finding a treatment center.

Visit Cure SMA
EveryLife Foundation for Rare Diseases logo

EveryLife Foundation for Rare Diseases

The foundation’s mission is to empower the rare disease patient community to advocate for impactful, science-driven legislation and policy that advances the equitable development of and access to lifesaving diagnoses, treatments, and cures.

Visit EveryLife
Global Genes RARE Foundation Alliance logo

Global Genes

A globally connected community committed to eliminating the challenges of rare disease by providing information, resources, and connections to all communities affected by rare diseases.

Visit Global Genes
Muscular Dystrophy Association logo

Muscular Dystrophy Association (MDA)

Transforming the lives of people living with muscular dystrophy, amyotrophic lateral sclerosis (ALS), and related neuromuscular diseases, through research, care, and advocacy. This organization can also help you find a treatment center.

Visit MDA
National Organization for Rare Disorders (also known as NORD) logo

National Organization for Rare Disorders (NORD)

NORD advances practical, meaningful, and enduring change so people with rare diseases can live their fullest and best lives.

Visit NORD

Join the ZOLGENSMA community

It can help to meet and join other SMA families. There are numerous social groups and organizations that would love to have you join the conversation. Here are some places to get started:

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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, during, 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, during, 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. at 1-833-828-3947 if a tumor develops.
  • Infusion-related reactions may occur during and after ZOLGENSMA infusion. Seek immediate medical evaluation if signs and symptoms of infusion-related reaction occur which may include rash, hives, vomiting, shortness of breath, respiratory symptoms, and/or changes in heart rate and blood pressure.

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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