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Navigating life with SMA after treatment

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SMA Atlas

Navigating life with SMA after treatment

Building our SMA care team


Building our SMA care team

Matt and Laura discuss the process of building Donovan’s SMA care team, which includes several different specialists they will mention, and what role they play on the care team as his parents.
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LAURA: We happen to live near a hospital that has an SMA clinic. And so we were able to find doctors that specialize in SMA pretty easily. When we were building our care team for Donovan, we started with our pulmonologist, who specializes in SMA, and so he was able to teach us some things about the disease.

For me, I think it’s, it’s okay to have people who are SMA experts and also other people that I just trust and I know. We have pretty developed relationships with those people that have known him over years. So you really do have a lot of personal interacting with those people.

MATT: It’s taken time to have the care team that we have today. The hard part about SMA, is that it’s a chronic, life-long illness.

As, as the parent, I’ve come to believe that it’s my job to be the quarterback for my son’s care. And I know Donovan better than any of his experts, but his neurologist knows neurology better than I ever will. And his pulmonologist knows pulmonology just as well as I know Donovan. So you need to have these other experts on staff, so to speak.

And so it’s really important to have these doctors and therapists involved. You’re human. You can’t know everything about everything.

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You may have heard the saying, “It takes a village to raise a child.” Truer words have never been spoken, especially for those raising a child with spinal muscular atrophy (SMA)—even after receiving treatment. Donovan, who was diagnosed with SMA Type 1, received ZOLGENSMA® (onasemnogene abeparvovec-xioi) when he was about 2 months old. While the treatment has given him a chance to grow, gain strength, and reach new milestones, he still has SMA. Over the past 4 years, Donovan has had his own village helping him. It includes different types of doctors, therapists, and other care providers, all working together to make sure he gets the care he needs.

Please see the Indication and Important Safety Information below and the accompanying Full Prescribing Information.

Building a care team after diagnosis

Donovan’s care team has grown since he was diagnosed with SMA. At diagnosis, Donovan’s parents, Matt and Laura, were still learning about SMA and what their son would need to successfully manage his disease. “As the parent, I’ve come to believe that it’s my job to be the quarterback for my son’s care,” shares Matt. “But I don’t believe it’s a realistic or even helpful expectation to have everything in place at diagnosis or even all at once. It will build over time.”

Fortunately, they live near a clinic specializing in SMA and could start building their team immediately. “The clinic has a number of specialists and makes referrals if they don’t have a certain resource available,” says Laura. It was through this clinic that Laura and Matt found a general practitioner of complex care. The doctor is familiar with complicated diseases like SMA and continues to be Donovan’s primary healthcare provider.

They also meet with a pulmonologist to help ensure Donovan is prepared with breathing support when he needs it. “We have a few machines to keep his lungs healthy,” Laura explains. “We use a bilevel positive airway pressure (BiPAP) machine when he is sick, which helps make breathing easier. His cough assist machine and a suction machine we use on a more regular basis. Mostly we use them when Donovan is feeling fatigued or we can tell he’s a little uncomfortable. We also use a nebulizer anytime he’s sick.” Additionally, Donovan sees an occupational therapist weekly to help him acclimate to new equipment as well as a physical therapist who focuses on getting him more mobile.

“It was helpful for me to know there was someone I could call if I needed help, or there was an important piece of information to have. But the care routine really does become second nature eventually.”

Laura, mother of Donovan who was diagnosed with SMA Type 1


Evolving the care team

As Donovan continues to grow, his care team evolves to support new demands. “It’s really important to have multiple doctors and therapists involved in his care team because he is growing and things manifest differently,” explains Matt. To address concerns about weakness in the muscles supporting Donovan’s spine, his parents added an orthopedist who uses massage-like techniques to provide comfort. To help ensure that Donovan gets the right amount of calories to manage his weight, a nutritionist monitors Donovan’s nutrition through a personalized plan. Donovan has a gastrostomy tube (G-tube) to help get those calories he needs. His parents also employ an in-home nurse. “She’s with him 8 hours a day and handles his day-to-day needs. It helps to have another experienced person on hand when we need to focus care, like when Donovan is sick,” explains Matt.

The best advice

Because no one knows what the future holds, Matt and Laura encourage other families to use all of the services and care that are available to them to give their child the best possible chance for the future. “It’s okay to have people who are SMA experts helping,” says Laura, “But it’s also important to have people who will work and listen to us because we know Donovan best. 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.”

Start building your care team today by working with your doctor to find the specialists that your child needs.

Malachi, who was diagnosed with SMA Type 1, in a gait trainer

Advocacy doesn’t stop after treatment

There is no expiration date on advocating for your child. Hear caregivers share the different ways they are still advocating for their children in and out of the home.

Donovan, who was diagnosed with SMA Type 1, playing with his dad

Preparing for the future

Hear the perspectives of multiple families about why it’s important to be knowledgeable about SMA and its management even after treatment.

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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 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.

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 AveXis at 833-828-3947.

Please see the Full Prescribing Information.