Watch family experiences with ZOLGENSMA

Stella’s story

Treated at ~1½ months old and filmed at 1½ years old

Watch Stella’s video

Lucy’s story

Treated at ~1½ months old and filmed at 1 years old

Watch Lucy’s video

Quinn’s story

Treated at ~15 months old and filmed at 2 years old

Watch Quinn’s video

Watch family experiences with ZOLGENSMA

Watch videos of families as they share their treatment journey, what the day of treatment with ZOLGENSMA® (onasemnogene abeparvovec-xioi) means to them, and how their child is doing after treatment. Each story is unique, but what they all have in common is a sense of hope and determination that will inspire you.

Results and outcomes vary among children based on several factors, including how far their SMA symptoms progressed prior to receiving treatment.


FAMILY STORIES

See how ZOLGENSMA is changing spinal muscular atrophy (SMA) Type 1 treatment

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Adalyne

SMA Type 1 (2 copies of SMN2 gene)
Age at treatment: ~2½ months old
Age in video: 3 years old

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Brady

SMA Type 2 (3 copies of SMN2 gene)
Age at treatment: 14 months old
Age in video: 2 years old

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Donovan

SMA Type 1 (2 copies of SMN2 gene)
Age at treatment: ~2 months old
Age in video: 4 years old

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Eli

SMA Type 1 (2 copies of SMN2 gene)
Age at treatment: ~4 months old
Age in video: 5 years old

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Evelyn

SMA Type 1 (2 copies of SMN2 gene)
Age at treatment: ~2 months old
Age in video: 4½ years old

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Louis

SMA Type 1 (2 copies of SMN2 gene)
Age at treatment: ~2½ months old
Age in video: 3 years old

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Lucy

SMA Diagnosed presymptomatically (2 copies of SMN2 gene)
Age at treatment: ~1½ months old
Age in video: 1 year old

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Maisie

SMA Type 1 (2 copies of SMN2 gene)
Age at treatment: ~20 months old
Age in video: 2 years old

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Malachi

SMA Type 1 (2 copies of SMN2 gene)
Age at treatment: ~4 months old
Age in video: 4 years old

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Matteo

SMA Type 1 (2 copies of SMN2 gene)
Age at treatment: ~1 month old
Age in video: 4½ years old

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Olivia

SMA Type 1 (2 copies of SMN2 gene)
Age at treatment: ~2½ months old
Age in video: 2 years old

Watch the video

Quinn

SMA Type 2 (3 copies of SMN2 gene)
Age at treatment: ~15 months old
Age in video: 2 years old

Watch the video

Stella

SMA Type 1 (2 copies of SMN2 gene)
Age at treatment: 1½ months old
Age in video: 1½ years old

Watch the video

Tenley

SMA Type 1 (2 copies of SMN2 gene)
Age at treatment: ~5½ months old
Age in video: 4½ years old

Watch the video

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.