Olivia’s story: Our experience with ZOLGENSMA

Olivia’s story

Meet Kirsten and Cody and their daughter, Olivia, who was diagnosed with SMA Type 1. Olivia was treated with ZOLGENSMA at 11 weeks old. See what she is doing at 2 years old in this video and hear her parents talk about the new movements Olivia has achieved.

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KIRSTEN: When Olivia was born, she appeared healthy, but between 2 and 6 weeks of age, she started to show signs of weakness and was not able to lift her head anymore or kick her legs.

KIRSTEN: When our pediatrician told us to go see a pediatric neurologist, we started doing our own research, and what we kept coming up with was spinal muscular atrophy. We were hopeful that’s not what she had because that meant that she would likely pass away before her second birthday.

KIRSTEN: When we met with the pediatric neurologist and Olivia was diagnosed with SMA, we were devastated.

CODY: We started looking at the different types of treatments. We read online, we reached out to medical providers to see what our options were.

KIRSTEN: Our pediatric neurologist told us that there was a treatment available and there were also clinical trials that were coming up, so we were excited to try those and see what would happen and to have some hope.

KIRSTEN: When it came to the clinical trial, my husband and I had some concerns that it may not work, as it was a trial and it was not an FDA-approved treatment, and we were wanting to make the best call for our daughter.

KIRSTEN: My husband and I decided to go with the clinical trial due to the results we saw from the first trial. And it was also treating the genetic root cause of SMA with a one-time dose.

CODY: I remember we were waiting to hear back to see if she was going to get accepted, everyone was staying by their phones, and when we got the call, we were both very ecstatic.

KIRSTEN: Prior to enrolling in the clinical trial, Olivia had to receive several blood tests.

KIRSTEN: After dosing she continued to do blood tests as well as several physical tests and swallow studies that were required as part of the trial.

KIRSTEN: Prior to her treatment, Olivia had to start an oral corticosteroid, and she stayed on that until her pediatric neurologist weaned her off of it.

CODY: Olivia received treatment with ZOLGENSMA in June 2018 when she was 11 weeks old.

KIRSTEN: I was just so happy once the IV was done. We were just so thankful and excited.

KIRSTEN: We celebrate Olivia's treatment day every year. It's a day where we celebrate what she's been through and what she has done since then. And it's a day that we remember the hope we had on that day and the hope we continue to have.

KIRSTEN: When Olivia was treated, SMA had taken away from her ability to move her legs. She could only move her arms from the elbow down.

KIRSTEN: Today Olivia is 2 years old, and she has gained so much since treatment.

KIRSTEN: When we saw all of the new movements Olivia was making, we were so excited to see that this treatment was working for her. We just felt like the sky was the limit.

KIRSTEN: She began kicking again, she was moving her arms more, and she was also starting to lift her elbow off of the ground as well.

KIRSTEN: Olivia is sitting up unassisted. She has her head control, she eats by mouth, she's not G-tube fed. Because Olivia cannot eat like a typical 2-year-old, her nutrition is supplemented with nutritional drinks.

KIRSTEN: She can breathe on her own. She doesn't have BiPAP. She has a weak cough and because of that, she uses a cough assist 2 times every day. She has a wheelchair that she can roll around in. And she can stand in a stander, and she just continues to get stronger and stronger.

KIRSTEN: We also have physical therapy, occupational therapy, and feeding therapy appointments on top of regular pediatrician appointments, neurologist appointments, pulmonologist appointments, nutritionist appointments, so she's a very busy girl, but that's what keeps her healthy and strong.

CODY: We hope in the future Olivia continues to be healthy and gain strength. We hope that she will be able to be independent one day. And we also hope that she’ll continue to work with her therapies and possibly be able to stand one day.

KIRSTEN: My hopes for Olivia's future are for her to be happy. I don't care if her life doesn't look like what people think it's supposed to look like. I want people to love her as for her and see her for all the amazing things that she is. She's the most beautiful, special little girl in the world. I want everyone to see that, too.

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Watch more stories like Olivia’s

Important Safety Information

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.

Important Safety Information

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 the patient experiences unexpected bleeding or bruising.
  • Thrombotic microangiopathy (TMA) has been reported to occur approximately one week after ZOLGENSMA infusion. Caregivers should 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 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.

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.