Every Day With SMA

Navigating life with SMA

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Every Day With SMA

Navigating life with SMA

Memorable moments

Celebrating special moments in your child’s life can give you a new appreciation for what is possible when living with spinal muscular atrophy (SMA) even after treatment. Hear how caregivers celebrate each moment in their unique way.

Stella, who was diagnosed with SMA Type 1, having a good hair day

Stella’s good hair day

Stella’s parents thought they might never experience the peskier parts of having children. Hear how Stella surprised them and made a memorable moment the first time she grabbed a fistful of her mom’s hair.

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

Slade’s first popsicle

Even though children with SMA can experience difficulties with eating and swallowing, Slade is making progress. Watch him taste the sweeter side of life in this video of him eating his first popsicle.

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

Brady goes mobile

Watch Brady’s parents recount the time he pulled himself to stand independently in his walker.

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 finds some newfound independence.

Ryker, who was diagnosed with SMA Type 1, in his wheelchair

Ryker turns 2

It's the happiest of birthdays for Ryker, who turned 2 years old! Watch a video of his parents reflecting on this joyous occasion while making lifelong memories.

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

Lucy’s sister shows the way

For Lucy, having a supportive sibling has made for many fun and memorable moments. Watch how these sisters work together to strengthen their bond.

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

Quinn proudly stands

Hear Joe and Annie recount the time their daughter, Quinn, received her leg braces and how that moment has shaped their hopes for Quinn’s future.

Natalie, who was diagnosed with SMA Type 2, coloring with a crayon

Natalie’s new move

The times when children are proud of themselves are heartwarming moments for parents. Watch Natalie’s parents talk about one such moment and the impact it had on their family.

Evelyn, who was diagnosed with SMA Type 1, traveling with her mom and dad

Adventures abroad with our child with SMA

When the Swiss Alps called, Evelyn answered. Read about the adventure her family never thought they would experience because of Evelyn’s SMA.

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.

Maisie, who was diagnosed with SMA Type 1, saying, “Momma”

Maisie says, “Momma”

Hear an SMA caregiver share the moment her daughter said, “Momma” and why that one incredible word meant so much to her.

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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.