Every Day With SMA

Navigating life with SMA

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Every Day With SMA

Navigating life with SMA

Stella’s good hair day


A stellar surprise

Hear SMA caregivers talk about the first time their daughter Stella tugged on her mom’s hair, an experience they thought they might never have.
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SAMANTHA: When Stella was first diagnosed, I remember being so upset that there was a mom complaining that their child was pulling their hair. And here I am with my 3-week-old baby, who changed my world already, that wasn’t able to even move her arms to do so. She wasn’t able to grasp anything at that point. So I never ever forget when I felt a tug on my hair. And at first I thought, “Oh, I’m caught on something,” but when I looked down, Stella had her hand wrapped around my hair and that was something she had never done before.

JEREMY: It made me emotional because it’s one of the very first times that Samantha and I together saw Stella make a significant movement physically.

SAMANTHA: I went from not having those experiences to being very thankful for the littlest experiences that we were having. To watch her do the littlest things, they mean so, so much to us and has given us another perspective on everything in life.

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Donovan, who was diagnosed with SMA Type 1, playing with his dad

Preparing for the future

Hear the perspectives of multiple families about why it’s important to be knowledgeable about SMA and its management even after treatment.

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 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.