Although Edan did not qualify for the clinical study, their pediatric neurologist thought he might qualify for treatment through the AveXis US Managed Access Program (MAP), which helps children with SMA get access to the investigational gene therapy treatment.
To his parents’ excitement, Edan qualified for the AveXis US MAP and received the investigational gene therapy treatment* in Madison, Wisconsin in 2019 when he was about 3 months old. “We had excellent doctors who helped take care of everything for us,” shares Rory. “We had to make our decision and focus on what was next after treatment.”
*Edan did not continue with another SMA treatment after receiving ZOLGENSMA.
Paying it forward
These days, Edan is an active little boy. “He’s constantly on the move, climbing and trying to escape to the park near our house,” shares Carolyn. “He’s very social. He will go onto the basketball court and try to play with the older kids. Even when we take him to the zoo, he’s more interested in the kids his age running around than the animals.” Rory says, “When he’s not outside, Edan is really inquisitive around the house. He’s always opening doors and cabinets, flipping through books, and exploring his world.”
Edan has been doing well thanks in part to the move to Minnesota, where newborn screening identified his SMA within days of his birth, leading to early treatment at only 2 weeks old. “A big part of the reason why SMA made it onto the newborn screening panel in Minnesota was due to one woman advocating for her child with SMA,” says Carolyn. “It used to be that parents advocated for newborn screening because their children were lost. We are now in a time where parents advocate because their children were saved.”
Since Edan’s SMA diagnosis and treatment, Rory and Carolyn have made advocacy an important part of their life’s work, endeavoring to get SMA on the newborn screening panel in all 50 states. In Wisconsin, where Edan received ZOLGENSMA, Carolyn wrote letters and made phone calls until she secured a meeting with the governor to advocate adding SMA to the state’s newborn screening panel. SMA was added to the Wisconsin newborn screening panel in October 2019. For his part, Rory has been in contact with legislators in his home state of Nebraska, and the issue of adding SMA to the state’s newborn screening panel is coming to the floor for a vote.
Rory and Carolyn recognize the power of coming together to help advance medicine, and understand that advocacy work is a bridge to help parents in the future who could benefit from early screening the way Edan has. For now, they continue to focus on feeling grateful for their son’s health, and for the chance to pay it forward.
Results and outcomes may vary among children based on several factors, including how far their SMA symptoms progressed prior to receiving treatment.
Watch videos about other families’ experiences with ZOLGENSMA and learn how other children are doing after treatment.
To learn more about newborn screening across the country and in your state, visit Cure SMA.
Indication and Important Safety Information for ZOLGENSMA® (onasemnogene abeparvovec-xioi)
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.
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 www.fda.gov/medwatch, or AveXis at 833-828-3947.
Please see the Full Prescribing Information.
The preceding article is content provided by our sponsor, AveXis. The views and opinions expressed in the content above are not the views and opinions of SMA News Today or its parent company, BioNews Services, LLC.
SMA News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified healthcare provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.
©2020 AveXis, Inc. All rights reserved. For US residents only. US-ZOL-20-0238 09/2020
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