Brady’s story: Our experience with ZOLGENSMA

Brady’s story

When Brady was diagnosed with SMA Type 2 at 13 months old, it was the kind of news no parent ever wants to receive. Hear how his parents, Nicole and Tyler, worked with their healthcare team through diagnosis and treatment with ZOLGENSMA and see how Brady is doing at 2 years old in this video.

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NICOLE: In July 2019, Brady was 13 months old and was diagnosed with SMA Type 2.

NICOLE: Our doctor was very sympathetic. She said, “Nicole, I’m so sorry, but I have to tell you this, but we have a plan.”

TYLER: They assured us that there's been some advancements, so that was a definite relief––still something no parent wants to hear ever, but there was some hope.

NICOLE: When we met with our genetic specialist and the pediatric neurologist, she actually had a slide show prepared for us. Through that slide show, she showed us what was missing. She went through all the types of SMA. She went through the different types of treatment. She said there are 2 FDA- approved treatments, and we chose ZOLGENSMA.

TYLER: We chose ZOLGENSMA because it was a one-time treatment and it treats the genetic root cause. The virus takes the new genes to the motor neuron to provide the missing proteins.

NICOLE: We went to the ZOLGENSMA webpage. We found videos of families just saying, “We went through the clinical trials. We did this….“ And we watched those videos so many times. We were confident in ZOLGENSMA because of the clinical trials. From the day we received diagnosis, our journey went really fast and our doctor was prepared.

NICOLE: The OneGene Program® called us after our doctor submitted the consent form. It was amazing to have them reach out so quickly because we felt like we had someone in our corner. Our Patient Resource Manager reached out to us right around the time OneGene did. She walked us through each step of the journey. She would call me and she would console me as a mother. She would say, “You’ve got this. We've got this. This is what comes next. This is what is the next step.”

NICOLE: Before Brady received ZOLGENSMA, we had to go through some tests to make sure that he qualified. We first did the antibody test then baseline tests. We began the corticosteroid with Brady the day before infusion and continued that afterwards. On treatment day, we were nervous on the way there, but our PRM was there waiting for us, and then that fear quickly went to excitement when we got to the hospital. I'll always remember the day that Brady received ZOLGENSMA.

TYLER: Brady received treatment in August 2019 when he was 14 months old.

NICOLE: When the infusion was over, I felt like I could breathe for the first time. We cheered. Our pediatric neurologist cheered. It was almost like a party. We had Brady his first party––his first ZOLGENSMA party. After treatment, Brady's liver enzymes were elevated. Our doctor had already been monitoring those, but once she received that back, she started monitoring those a little closer.

TYLER: At the time of treatment with ZOLGENSMA, Brady had lost ability in his legs. He was struggling to crawl. He was struggling to hold his sippy cups, hold his toys. He had lost a lot of mobility.

NICOLE: Today, Brady can climb the stairs. He can get in his walker independently. He can walk across the yard with his walker. Brady eats on his own. He uses utensils and finger foods and he eats everything by mouth.

MASON: Me and Brady like to play catch and ride horses, go fishing together. I help Brady by getting him on the four-wheeler, helping him get up the stairs, and helping him cast his fishing pole.

TYLER: He is a wild child. He never stops, he never slows down from the time he gets up until the time he goes to bed. He is going 100 miles an hour, never stops talking. He amazes me every day with the new things that he's doing.

NICOLE: Brady hasn't needed breathing therapy. We currently do physical therapy and hippotherapy. ZOLGENSMA has exceeded our expectations. We wanted to stop progression of the disease, and we are not only seeing that, we're seeing Brady continue to get stronger.

NICOLE: Like any parent, my hope for Brady's future is that he continues to grow. SMA hasn't changed that hope. I have the same hope for Brady as I do Mason. I want him to continue to be a good person, I want him to continue to be healthy and I want him to continue to grow.

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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.
  • 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.
  • 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. Contact the patient’s doctor immediately if you see 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 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.