Every Day With SMA

Navigating life with SMA

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Every Day With SMA

Navigating life with SMA

Ryker turns 2


A reason to celebrate

Join in the excitement of Ryker's 2nd birthday celebration and hear his parents reflect on what a joyous occasion it is for the entire family.
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STEPHANIE: Ryker just turned 2 years old, and this is such a huge milestone for us and for SMA kids because without treatment, Type 1 kids are only given until 2 years old to live.

STEPHANIE: The reason that it’s so memorable for us is because my daughter Aizlynn was born in 2012 and there were no treatments for SMA.

GLEN: Ryker and Aizlynn, they both have 2 totally different stories. And through the pain and the heartache, we’ve found the ability to find grace, beauty, love, all sorts of amazing things that we just would have never ever experienced. The hope and the new possibilities that are there is just incredibly exciting.

STEPHANIE: My hopes for Ryker’s future are that he continues to develop the way that he is. I would love to see him be able to walk without assistance. One of my biggest hopes is just to be able to see him grow up and graduate high school and get married—and those were things that we didn’t get to see our daughter do.

GLEN: But you don’t know what’s gonna happen tomorrow. So take it a day at a time and just love, you know, those children, the absolute best that you can and let them experience life the best way that they can.

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Lucy, who was diagnosed with SMA presymptomatically, standing with assistance from her older sister

Lucy’s sister shows the way

For Lucy, who was diagnosed with SMA through newborn screening, having a supportive sibling has made for many fun memorable moments. Watch how these sisters work together to get Lucy up and walking with assistance.

Brady, who was diagnosed with SMA Type 2, standing in his walker

Brady goes mobile

Watch the first time Brady, who was diagnosed with SMA Type 2, pulled himself to stand independently in his walker.

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.