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It Takes a Village

It Takes a Village

You may have heard the saying, “It takes a village to raise a child.” Truer words have never been spoken, especially for those of us raising a child with spinal muscular atrophy (SMA)—even after receiving treatment with ZOLGENSMA® (onasemnogene abeparvovec-xioi). Over the past couple of years, our village has included many doctors, therapists, and other care providers, all working together to make sure Donovan gets the care he needs.

Donovan was treated with ZOLGENSMA when he was 2 months old. While the treatment has given him a chance to grow, gain strength, and reach new milestones, he still has SMA. To help Donovan grow and manage his SMA, we had the help of a care team that understood his needs.

Indication

ZOLGENSMA® (onasemnogene abeparvovec-xioi) 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.

Important Safety Information

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.

Please see additional Important Safety Information below and accompanying Full Prescribing Information.

Building a care team after diagnosis

Our care team was at its largest and most varied the year after we got Donovan’s diagnosis. We were still learning about SMA and figuring out what Donovan needed. We started working with a clinic specializing in SMA near where we live. This clinic had a number of specialists and would make referrals if they didn’t have a certain resource available. In fact, it was through this clinic that we found a general practitioner of complex care. This doctor is familiar with complicated diseases like SMA and continues to be Donovan’s primary healthcare provider today.

“In the beginning, a lot of people were involved in Donovan’s care. It was very important for us because, while we didn’t always know who to ask what, just being able to ask someone helped us get to the right person.”
Laura, mother of Donovan who has SMA Type 1

We also met with a pulmonologist to help ensure Donovan was prepared with breathing support when he needed it. While Donovan does not have a daily respiratory routine, we have a bilevel positive airway pressure (BiPAP) machine that helps make breathing easier as well as a cough assist machine. We also have a respiratory vest for suction, as needed.

To help ensure that Donovan was getting the right amount of calories to manage his weight, we met with a nutritionist who taught us how to use Donovan’s gastrostomy tube (G-tube) and suggested foods to feed him by mouth. Feeding therapy was also offered, so we gave it a try. Even though Donovan still has a feeding tube, we encourage him to eat by mouth.

Adapting our care team over time

As Donovan grew from an infant to a toddler, I became more and more concerned about scoliosis because of weakness in the muscles that support his spine. So we started working with an orthopedist. Over time, we added an osteopathic medicine doctor to our care team. She uses massage-like techniques to relieve muscle tightness. Between appointments we use a stander to help maintain muscle strength in Donovan’s legs and increase his range of motion.

Donovan also works with a physical therapist and an occupational therapist once a week. He saw a speech therapist but has since graduated from the program. Throughout the course of this year, we consulted with a social worker and tried other types of therapy, including aquatic (which Donovan loves), art, and music.

As Donovan grows, we will likely face new challenges. Physically, mobility will become more of an issue. We will need interventional support at school and occupational therapy will be needed to make sure he can complete certain daily tasks on his own.

The best advice

Because no one knows what the future holds, I encourage other families to use all of the services and care that are available to them now to give their child the best possible chance for their future.

It may seem like a lot of people are involved, but when you’re dealing with SMA, having a lot of minds working together to solve problems, offer solutions, and suggest resources is essential.

Watch videos and learn from families who have experience with SMA at ZOLGENSMA.com.

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

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 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 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.

©2019 AveXis, Inc. All rights reserved. US-ZOL-19-0217 09/2019

Indication

ZOLGENSMA® (onasemnogene abeparvovec-xioi) 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.

Important Safety Information

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.


Please see additional Important Safety Information below and accompanying Full Prescribing Information.