Posts by: AveXis

Paying it forward. Meet Regina and Al. Their friendship and respect for one another all started at a fundraiser before a Phillies baseball game in 2019. “I watched Regina give a beautiful speech at the event about her recently diagnosed son, Shane," recalled Al. “I thought to myself, ‘My friends at Cure SMA need to meet this amazing mom and get her involved.’” Al knew a thing or two about connecting people with the SMA community. His involvement started more than 25 years ago when his son, Jack, was diagnosed with spinal muscular atrophy (SMA) Type 1. At the time, there were no treatment options, and there was very little hope. Encouraged by Audrey Lewis, the founder of Cure SMA, Al attended his first Cure SMA conference in 1996. “Audrey supported me when I felt very scared and disoriented. She and so many others at Cure SMA helped me to know that I wouldn't be alone,” said Al. Since then, he has used his doctorate in child family psychology to facilitate dozens of workshops to help families in the SMA community. When he met Regina, he saw someone to whom he could pay forward the support that he received from Audrey and others when Jack was diagnosed. Regina’s son Shane was diagnosed with SMA Type 2 in 2018 when he was 10 months old. At the time of diagnosis, there was only one FDA-approved treatment available, which Shane was treated with up until ZOLGENSMA® (onasemnogene abeparvovec-xioi) was approved in May 2019. He was infused with ZOLGENSMA a few months later when he was about 1½ years old. Following Shane’s diagnosis, Regina became actively involved in the SMA community, and that’s when she first met Al. “He came up to Shane and me after my speech at the fundraiser before the baseball game. He was the first parent we had ever met who has a child with SMA,” recalled Regina. As they talked and shared their experiences with SMA, Shane started hysterically crying. “I’m trying to have a conversation with this guy who I just met, and Shane is crying so loud you can’t hear anything. And Al was like, ‘Wow, what a beautiful sound,’ because he had never heard his son, Jack, cry that loud,” said Regina, “And that really put things into perspective for me. How lucky are we to experience it, and for it to be so different because of treatment.” 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 a 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. Finding inspiration and gaining an appreciation. Even with more than 23 years separating their SMA experience, Regina and Al continue to find inspiration in each other. “I knew in my heart that Regina would follow a path similar to mine. She can be a voice for other families who may not be as comfortable standing up in front of a room full of people, and she can get things done. Look at what Regina has already accomplished with newborn screening. She used her experience as an attorney, she brought people together, and got newborn screening passed in New Jersey. She has already made a huge difference,” explained Al. And Regina is quick to pay the compliment back, “He has taught me to appreciate absolutely everything that Shane is and will be. That’s the way Al lives his life being Jack’s Dad, and how he makes the SMA community better,” shared Regina. “I’m trying to emulate him so that I can be a better person for my son and the SMA community. It really is beautiful, and it is a gift.” “Regina’s family represents the hope that we didn’t have back in 1995. I never imagined in Jack’s lifetime, or mine, that science would catch up with our kids and help them get stronger.” - Al, father of Jack who has SMA Bridging decades of SMA experiences. Regina and Al’s connection extends beyond their SMA relationship. It also involves their children. It was at a Cure SMA Walk-n-Roll event that Jack first met Shane and his family. “Jack loves these events because it’s where he can see other people like him,” said Al. “And I had explained to Jack about ZOLGENSMA and about meeting Shane’s family. It was also cool for him to understand that Shane has SMA, but he is stronger because of treatment. Because of this event and meeting Regina, Jack now considers Regina and Shane his buddies, and he talks with them regularly. Regina remembers that first encounter vividly. “We brought Shane over to meet Jack. He has an aura about him, and everyone wants to be around him,” said Regina. “Recently, we had so much fun recording a Happy Birthday message and sending it to Jack for his birthday. It’s a connection that’s wonderful for both of our families.” As they look to the future, Al and Regina feel hopeful and excited, not just about treatment, but also by the spirit of the people in the SMA community. “I call us the luckiest of the unlucky,” explained Regina. “We have this difficult diagnosis, but our community is doing amazing things together and there are treatment options now. There’s hope. There’s so much out there that we have to be thankful for. My message to everyone is just never give up.” As new generations join the SMA community, it’s exciting to know that they have a vast network of people like Al and Regina to connect with and be inspired by. Watch videos about other families’ experiences with ZOLGENSMA and learn how their children are doing after treatment. Results and outcomes vary among children based on several factors, including how far their SMA symptoms progressed prior to receiving treatment. Indication and Important Safety Information for ZOLGENSMA® (onasemnogene abeparvovecxioi) 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-20-0141 07/2020

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Posts by: AveXis

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The importance of newborn screening for SMA Sponsored Post

How different generations living with SMA can teach one another