Matteo’s story: Our experience with ZOLGENSMA

Matteo’s story

Hear from Nicole and Derwin, whose son, Matteo, was diagnosed with SMA Type 1 and was treated with ZOLGENSMA at about 1 month old. Follow Matteo over the years and learn about what he wants to be when he grows up now that he is 4½ years old in this video.

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DERWIN: The first time I heard about SMA, my sister was pregnant and her doctor tested her for SMA. Her doctor told her there’s a chance your relatives are carriers as well. So, when we decided that we wanted to have children, Nicole actually went to her doctor and she told us that we should get tested. And we set up an appointment for two weeks, and between those two weeks, she ended up pregnant. She got tested and on my birthday, we got the results that she was a carrier.

NICOLE: We test him. We find out he’s a carrier. So, we had an amnio done. I got a call one day at work. It was, the doctor and she wanted us to come in. I knew it. (laughs) I knew right away that it wasn’t good news.

DERWIN: Nicole was 5 months pregnant when Matteo was diagnosed with SMA. It’s so tough to think that this little thing that you haven’t even met in person; you are so attached to them. You love them so much. It’s just, it hurt. We cried. It hurt some more. But at the same time, we knew we couldn’t just give up on him. My wife got in touch with Cure SMA. They put us in touch with other parents. And, one of the parents guided us in the two options that we had.

NICOLE: We decided on this treatment, because it was a one-time treatment. It actually replaces the missing gene. So that meant a lot to us. Matteo received treatment when he was 27 days old.

DERWIN: I’ll always remember that one day.

NICOLE: Matteo is your typical three-year-old. He’s fun. He’s outgoing and he’s very social. When we decided to go with this trial, we really didn’t have many expectations. I wanted him to be able to breathe on his own and maybe to be able to eat on his own.

DERWIN: Him walking, him running, him doing everything that he’s doing now, is just a bonus.

NICOLE: We don’t need to do anything to manage Matteo’s SMA at this point.

MATTEO: Hi, my name is Matteo. I’m 4½ years old. I want to be a chef astronaut. And I want to cook on the moon.

DERWIN: Matteo has become an awesome little human. He’s very loving. He’s very caring. But he’s also very headstrong and he knows what he wants. And it’s funny to see how someone that is so tiny can grow and show such personality.

NICOLE: Matteo can walk. He can run. He can jump. He can climb up the stairs. He can play in the playground. He can eat. He can talk. He’s very vocal. He received gene therapy at 27 days old, it really did change his prognosis.

DERWIN: From the moment he wakes up to the moment he goes to sleep, he can outlast all of us. And he just goes, goes, goes. He’s just a fun boy. He doesn’t know anything else other than fun.

NICOLE: Matteo’s in preschool and he goes to school Monday through Friday. Seeing Matteo walk into school was something that the doctors told us would probably never happen.

DERWIN: We’re very happy with the opportunity that we’ve had to see Matteo grow. From where we were at, where we had no options or no hope, basically, to where we are today.

NICOLE: I think it’s important to share our story because it gives people hope. It shows people that the treatment is working. It helps parents who were just newly diagnosed and are drowning in this diagnosis. It’s great to have a community that can help you out and that you can talk to and understands what you’ve really gone through.

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Important Safety Information

What is the most important information I should know about ZOLGENSMA?

  • ZOLGENSMA can increase liver enzyme levels and cause acute serious liver injury or acute liver failure which could result in death.
  • 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 increase liver enzyme levels and cause acute serious liver injury or acute liver failure which could result in death.
  • 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, if the patient misses a dose of corticosteroid or vomits it up, or if the patient experiences a decrease in alertness.

What should I watch for before and after infusion with ZOLGENSMA?

  • Infections before or after ZOLGENSMA infusion can lead to more serious complications. Caregivers and close contacts with the patient should follow infection prevention procedures. Contact the patient’s doctor immediately if the patient experiences any signs of a possible 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 the patient experiences unexpected bleeding or bruising.
  • Thrombotic microangiopathy (TMA) has been reported to generally occur within the first two weeks after ZOLGENSMA infusion. Seek immediate medical attention if the patient experiences any signs or symptoms of TMA, such as unexpected bruising or bleeding, seizures, or decreased urine output.

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 influenza and respiratory syncytial virus (RSV) is recommended and vaccination status should be up-to-date prior to ZOLGENSMA administration. Please consult the patient’s doctor.

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 patient body waste for one 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 Novartis Gene Therapies, Inc. at 833-828-3947.

Please see the Full Prescribing Information.