Every Day With SMA

Navigating life with SMA

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Every Day With SMA

Navigating life with SMA

Brady goes mobile

A new first

Hear from SMA caregivers about how their son surprised them by pulling himself to stand independently in his walker.
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NICOLE: A memorable time was when Brady pulled to stand independently on his walker. In true Brady style, Brady pulled up to his walker without any assistance when no one was expecting it. He did it and he stared at us like we were crazy, like he’d been doing it forever.

TYLER: That was something we had been working on for months and months for him to be strong enough to stand up in his walker and walk across the house on his own. That was such a huge milestone, something that we had wanted from the very beginning.

NICOLE: Being able to get into his walker on his own and to take off where he wants to go, was, it opened up Brady’s world. He can now get to the ball if he wants it or go to the sandbox if he wants to. And it’s also opened our world up, where we have to chase him more, but we’ll take it.

NICOLE: He just does it like it’s no big deal now. Like he’s been doing it forever and he’s surprised that we’re excited every time now. He rolls his eyes and goes on about his way.

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Quinn, who was diagnosed with SMA Type 2, standing assisted at a table

Quinn proudly stands

Achieving a new milestone in SMA is always cause for celebration. Hear Joe and Annie recount the moment their daughter, Quinn, received her leg braces and was able to stand for the first time.

Olivia, who was diagnosed with SMA Type 1, in her wheelchair

Olivia discovers her independence

Hear about Olivia as she gets her first wheelchair, discovers her world, and explores some of her newfound independence in this video.

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.