Donovan’s story: Our experience with ZOLGENSMA

Donovan’s story

Meet Laura and Matt and their son, Donovan, who was diagnosed with SMA Type 1 at about 1 month old and was treated with ZOLGENSMA at about 2 months old. Follow Donovan over the years since treatment and see what he is up to as a 4-year-old in this video. You’ll hear from his proud parents and about his excitement for starting preschool.

Read the transcript

LAURA: We have 2 kids. We have a daughter named Elizabeth and then we have our son, Donovan. Donovan was born with SMA Type 1. When he was born he had normal scores. He had normal delivery. When we went to his four-week checkup, he wasn’t responding normally. And the pediatrician said that he was very floppy, that something wasn’t right. At diagnosis, we were told that Donovan would continue to lose any strength that he did have, to move, and then he would also lose his ability to swallow and, eventually, to breathe. We met a doctor who had been working on the clinical stages of a treatment for SMA.

MATT: And she’s like, you need to know that there’s a trial going on.

LAURA: We were told that it’s a single dose through an IV. We thought, how can we not try? How can we not give him a chance? On the treatment day, we were excited, we were hopeful. We were so happy that today we are making a difference.

MATT: I will always remember the day that Donovan was treated.

LAURA: After treatment I saw little changes.

MATT: I remember he was laying there on the changing table and he, he couldn’t move at this point. And he takes his arm and he goes like this and he like moves his forearm and his wrist up just a little bit. And that was the first time that we saw like the tide had turned against SMA a little bit.

LAURA: Donovan is 2 and a half years old and he can roll. Donovan can sit up independently. He can hold his head up. We’re actually able to see this treatment working as he is growing and getting to be himself at home, with our family, spending time together. He does have symptoms of SMA. But he’s also growing stronger every day. And he is a very happy kid.

DONOVAN: I am Donovan and I’m 4 years old. I’m so excited to start school. When I grow up, I want to be a scientist.

LAURA: Donovan’s doing great. He’s a smart kid. Donovan has a really full life.

MATT: Since treatment, his abilities have grown a lot more than, than we had expected. We didn’t really know what to expect. He’s talking. He doesn’t stop talking. Which is a, another kind of major thing. Not everyone with SMA Type 1 is verbal.

LAURA: He has really good fine motor control. He can manipulate toys. And he can feed himself. He can talk. He is learning to read. And he can sit up by himself all day long. He can push his manual wheelchair. Donovan can do most activities that 4-year-olds can do. Even though Donovan’s received treatment, SMA does still affect him today.

MATT: I think that there’s a, a big draw to only talk about the positive. Especially when you’re, you’re so grateful for what he’s gotten. But it hasn’t erased the disease.

LAURA: We go to an SMA clinic and we will see a neurologist. We see a pulmonologist. We see a dietitian. And we see an occupational therapist at clinic that helps us with equipment. He also sees a physical therapist regularly. Getting treatment for SMA is kind of like step one. And then your whole life is ahead of you. So, you want to make sure that your child has everything that they need to grow and thrive.

MATT: It’s cool that we get to think about later in life. Which is not something you think about with SMA Type 1 because until recently, there was no treatment for it. But we get the chance to do that now. Having ZOLGENSMA has completely changed the trajectory of his life.

Close the transcript

Watch more stories like Donovan’s

Matteo’s story

Treated between 1-3 months

Louis’ story

Treated between 1-3 months

See all videos

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.