Connect with the SMA community

Remember that you are not alone during your journey with spinal muscular atrophy (SMA). There are many advocacy organizations and resources that help educate, support, and connect families like yours.

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Connect with the SMA community

Remember that you are not alone during your journey with spinal muscular atrophy (SMA). There are many advocacy organizations and resources that help educate, support, and connect families like yours.

Maisie, treated at ~20 months and pictured at 2½ years, was diagnosed with SMA Type 1.

Cure SMA

Cure SMA is leading the way to a world without SMA, the number one genetic cause of death for infants. They fund and direct comprehensive research that drives breakthroughs in treatment and care, and they provide families the support they need for today. Learn more at CureSMA.org.

EveryLife Foundation for Rare Diseases

EveryLife Foundation for Rare Diseases is dedicated to advancing the development of treatment and diagnostic opportunities for patients with rare diseases through science-driven public policy. They provide training, education, resources, and opportunities to make patients’ voices heard to help change public policy and save lives. Learn more at EveryLifeFoundation.org.

Global Genes

Global Genes educates, equips, and empowers the global community with tools and resources to influence change. They connect rare disease patients, advocates, and rare disease experts to help eliminate the challenges of rare disease. Learn more at GlobalGenes.org.

Muscular Dystrophy Association (MDA)

MDA is committed to transforming the lives of people affected by muscular dystrophy, amyotrophic lateral sclerosis (ALS), and related neuromuscular diseases through innovations in science and innovations in care. MDA’s legacy of progress has always started with families at the heart of all they do. The victories of their past will lead to tomorrow’s treatments and cures. Learn more at MDA.org.

I became active in the SMA community when Maisie was diagnosed with SMA Type 1. I wasn’t sure in what aspect, but I knew we would need them. Ciji, mother of Maisie

National Organization for Rare Disorders (NORD)

NORD is a patient advocacy organization dedicated to individuals with rare diseases and the organizations that serve them. NORD, along with its more than 280 patient organization members, is committed to the identification, treatment, and cure of rare disorders through programs of education, advocacy, research, and patient services. Learn more at RareDiseases.org.

Additional support and education

Online and other social media sites can be great places to find SMA communities. Search for SMA communities and discover numerous groups dedicated to helping families and sharing experiences.

Meet Malachi

Hear from Malachi's family who have firsthand experience with ZOLGENSMA.

Watch Malachi's video

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.