How different generations living with SMA can teach one another
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.
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