Let’s be honest, spinal muscular atrophy (SMA) is overwhelming, confusing, and scary for everyone. We were shocked and heartbroken the moment we were told our son, Malachi, then 3 months old, has SMA Type 1. So much was happening as we processed this devastating news—from being told he would only have 6 to 12 months to live to there is no cure or approved treatment. All we could think about was losing our little boy.

Within days of the diagnosis we discovered a clinical study for an investigational gene therapy for SMA Type 1. At about 4 months old, Malachi was treated with ZOLGENSMA^® (onasemnogene abeparvovec-xioi) as part of the clinical study. After the treatment was complete, we were hopeful. With time, we watched it begin to work. It helped Malachi gain the strength he needed to grow stronger and to keep his SMA Type 1 from getting worse. Even after treatment, we knew that Malachi still had SMA and we needed to focus on managing it. Were we nervous about this next chapter? Yes, but our confidence about living with SMA was growing after Malachi received treatment.

Finding support through connections

When Malachi was diagnosed, we hadn’t heard of SMA and we didn’t know anything about it, so we started reaching out to other families who had experience with this disease. We joined numerous Facebook groups and spoke with families who have children with SMA to ask questions and learn more. It meant a lot to us to talk to and meet with other parents of children with SMA because we could relate to each other and there’s a sense of openness, understanding, and comfort. And while physicians know a lot about the medical side of SMA, talking with other parents about the daily needs of a child with SMA has helped us understand Malachi’s needs at home. Every conversation and correspondence with these families who had walked in our shoes made us stronger and more informed.

After treatment, we reached out to the SMA community again to learn about managing SMA. We gained more confidence as we met others in the community and began building Malachi’s healthcare team, including specialists and therapists. Their advice, support, and insights really helped us understand the importance of managing Malachi’s SMA and helped him enjoy each day to the fullest. Today, he is zooming around in his wheelchair, feeding himself, and becoming more independent.

“Connecting with families who already had experience with SMA gave us a sense of security and the confidence that, yes, we can do this.”
-Tina and Torence, parents of Malachi who has SMA Type 1

Never stop learning

It’s important for us to stay current and knowledgeable about SMA so we can give Malachi every opportunity to live life to the fullest. While he doesn’t need to use multiple machines to help manage his SMA, we have taken the time to learn about the different types of medical equipment a child with SMA may need because, down the road, he could need more support. Also, learning about assistive devices and equipment gives us an opportunity to help others. People reach out to us on a regular basis to ask questions, especially newly diagnosed families. We just keep learning everything about the world of SMA in order to help our own family and other parents of children living with SMA as their children grow and their needs change. If you have any medical questions, you should always talk to your child’s doctor first.

The biggest piece of advice we have for newly diagnosed families or those who have already gone through a treatment is to have patience. At first you will feel overwhelmed and unknowledgeable about everything, but you don’t have to learn everything in the first hour. Be proactive, but don’t overwhelm yourself with too much information. Definitely give yourself a moment to grieve and process, and then jump into motion to start finding treatment and advocating for your child.

“Act fast on choosing a treatment but have patience with learning about SMA. Don’t overwhelm yourself.”
-Tina and Torence, parents of Malachi who has SMA Type 1

Our lives today

Malachi does physical and occupational therapy nearly every day at a therapist’s office and we do some at home, too. Twice a year we go to Nationwide Children’s Hospital in Columbus, Ohio, for additional physical and occupational therapy. They teach us new things we can do with Malachi that we incorporate at home. We also use a cough-assist machine when Malachi is sick with an upper respiratory infection and to help prevent pneumonia.

Caring for yourself and other family members

Lastly, always take time for you and your spouse. And while it’s important to carve out quiet time just for the two of you, you should also take breaks separately. Everyone needs his or her own space, and it helps with keeping your mind in a positive place. If you can, find someone you trust to watch your child, even if it’s just for an hour to give you a break. We have found that taking time to run an errand, go for a walk, or grab a coffee with a friend has huge mental and physical benefits.

If there are other children in your family, spend quality time alone with them. Whether it’s taking them to a movie or grabbing a pizza together, letting siblings feel special and important helps reinforce that every member of the family is loved equally.

Your SMA journey will be filled with ups and downs. But when you’re rested, mentally prepared, and working together, you’ll feel more confident and ready for the next step in your journey.

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

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.

• 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.

• 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.

• 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.

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