Every Day With SMA

Navigating life with SMA

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Every Day With SMA

Navigating life with SMA

Olivia discovers her independence

The world gets bigger

Hear about Olivia's experience getting her first wheelchair and how that sparked her curiosity and a desire for independence.
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KIRSTEN: A memorable moment is getting her first wheelchair and watching her figure it out and discover the world around her. As an opinionated 2-year-old, those are really important things to be able to do.

CODY: She’s able to go out and explore on her own, it gives her independence. She’s able to get into trouble, which is actually a good thing.

KIRSTEN: If she needs to go one way, I’m trying to push her one way, she’ll put the brakes on and just hold onto her wheel so that she doesn’t have to go anywhere.

KIRSTEN: She will leave the room that we’re in, and I think it’s because we’re around her so much, and so she's like, “Finally, I can get some peace and quiet by myself and I don't have to be around you people all the time.”

KIRSTEN: It’s just watching her be her opinionated self and be able to move around on her own and do all these things—it’s amazing.

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Slade, who was diagnosed with SMA Type 1, laying on a blanket and eating a popsicle

Slade’s first popsicle

Watch Slade taste the sweeter side of life in this video commemorating his first popsicle.

Matteo, who was diagnosed with SMA Type 1, walking with his mom and dad

Matteo’s first day of school

Hear an SMA family describe the moment their son walked into class on the first day of school and why it means so much to them.

Preparing for the future

Check out resources and advice from other caregivers to help you navigate your child’s future with SMA.

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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.

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.

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 833-828-3947.

Please see the Full Prescribing Information.