Learn how Slade is doing after treatment

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Full Prescribing Information

Important Safety Information

Slade, who has SMA Type 1, with his parents and older brother

Daryn and Morgan with their son Slade, who has SMA Type 1, and his older brother Callan.

Celebrating “Inchstones” in SMA

When Morgan and Daryn received the news that their son Slade has spinal muscular atrophy (SMA) Type 1, they were overcome with shock as the doctor explained that this rare disease was fatal without treatment. They immediately started doing their research and discovered 2 treatment options for SMA. One of them, called ZOLGENSMA® (onasemnogene abeparvovec-xioi), had just been approved by the FDA a few weeks prior.

“After careful consideration and discussions with Slade’s doctor, we strongly felt that ZOLGENSMA was the best option for us. The treatment was a gene therapy given one time only,” said Morgan. And when Slade was almost 7 months old, he received ZOLGENSMA.

Now 13 months old, Slade is continuing to make progress and Morgan and Daryn are grateful for every day they have together as a family. “We like to focus on Slade’s inchstones, not milestones, because every little improvement is a huge win for him,” said Daryn.

Learn more about Slade’s SMA story from Morgan’s perspective, including what he can do after treatment and why they chose ZOLGENSMA.

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

Read Slade’s story

Indication and Important Safety Information


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 the vein. ZOLGENSMA was not evaluated in patients with advanced SMA.

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 a patient experiences unexpected bleeding or bruising.

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.

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, or AveXis at 833-828-3947.

Please see the Full Prescribing Information.


The ZOLGENSMA Team at AveXis

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AveXis, Inc.

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Bannockburn, IL 60015


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US-ZOL-20-0018 02/2020

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