Malachi’s story: Our experience with ZOLGENSMA

Malachi’s story

Hear from Tina and Torence, whose son, Malachi, was diagnosed with SMA Type 1 and was treated with ZOLGENSMA at about 4 months old. Their story begins with heartbreak and turns into one filled with joy. Follow Malachi over the years and see what he is up to as a 4-year-old in this video.

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TINA: Malachi was born with spinal muscular atrophy Type 1. The first symptoms that we saw with Malachi was that he was no longer lifting his legs or his head like he did at birth. So when we took him to the doctor, I knew it wasn’t going to be good.

TORENCE: Diagnosis day. There wasn’t really much that they told us. “Take him home and love him, and you’d be lucky enough to have him a year or two.” And there was pretty much nothing that we were given that would have been hopeful.

TINA: The momma bear in me said to the doctor that my son would not be a statistic. And I had to hold tight on that. I went online and was searching for some information about SMA and what that meant for our lives, and I came across a clinical trial. We found the treatment intriguing because it was replacing the defective gene. Malachi at the time of treatment was very weak. He could barely move his hands and his feet. He was not able to lift his head, hands, arms, legs. Malachi was given treatment when he was 4 months old and two days. I’ll always remember that one day. It was a refreshing day. We had a lot of hope to look forward to the future.

TORENCE: After treatment, we felt excited to see what would happen next. Seeing just the slightest movement in his feet gave us real hope.

TINA: He just was gaining more and more strength. He was moving his hands. He started lifting his legs up, and within 2 months he was trying to roll. Since treatment 2 years ago, Malachi has been able to roll, lift his legs in the air freely, lift his arms. He is mobile. He’s able to move himself, gets to point A to point B. He’s very vocal. He was not very vocal prior. And he feeds himself, and that to me is a huge goal. He does not rely on us to do everything for him.

TORENCE: SMA has just changed my outlook on our family.

TINA: It makes you want to take every milestone that the kids do and you just hold on to it that much. Like everything means more now. When Malachi does anything new, it’s very exciting.

MALACHI: I’m Malachi and I’m 4 years old. I like to play basketball. When I grow up, I want to be a firefighter.

TINA: Since treatment, Malachi has gained a lot of strength. He went from barely moving to being able to stand against a chair. He goes to school part time. He loves it. He likes to play games. He plays baseball in gym and kicks balls and reads books and sings along to the teacher’s songs. And I mean these are things that we were told we would never see. Malachi likes independence. He likes to try things on his own and he will try it for a long time before he asks for help. SMA still limits him. He’s not able to walk. He’s not able to run. So he’s not like a typical child where he can just get up and go do what he wants getting dressed, brushing his teeth. He’s learning, but still needs quite a bit of assistance to get through his daily tasks. Managing Malachi’s care today goes with a lot of teamwork. It’s his father and I, of course. And then we have a team of therapists that we work very closely with.

TORENCE: SMA impacts Malachi’s nutrition and growth. He’s actually just now starting to get on the growth chart at 4 years old, which we’re really excited about. He notices that he’s different, but he doesn’t let that stand in his way. Every day is a blessing, no matter what happens. Whether it’s good or bad. It’s truly something that you can learn from.

MALACHI: Never give up.

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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.