Adalyne’s story: Our experience with ZOLGENSMA

Adalyne’s story

When Patti and Brandon’s first daughter was diagnosed, they knew nothing about SMA. Hear how their journey was different with Adalyne, their second daughter, who was treated at about 2½ months and is 3 years old in the video.

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PATTI: My daughter Adalyne, Addy, has SMA Type 1. Our first daughter Madison was born back in 2004 and at that time we knew nothing about SMA. Unfortunately, the disease really was tough on her body and at the age of nine months, she was G-tube fed and by the time she passed at 3½, she was unable to move any part of her body except for her finger. Maybe because I was a little paranoid, I was watching early on. With Addy, I really started noticing a lot of the same symptoms that Madison had.

BRANDON: She had considerable head lag. You could tell that her, you know, her extremities were definitely getting weaker. It was almost instantaneous where she was able to quit moving her legs.

PATTI: We decided to go to the doctor with Addy around six weeks of age. We went and had her tested. Our pediatrician really felt like it was gonna come back negative. It took about a week. She just happened to be working the next day on a Saturday and happened to tell her nurse to go check and see if anything had come in, not expecting that it had, and it had come in. And she said she was quite devastated herself.

BRANDON: Addy was diagnosed in September of 2015.

PATTI: Our pediatrician knew that we were pretty advanced in knowing people who knew what’s going on out in the world, and the first thing she said is “start doing your research.”

BRANDON: I found several different clinical trials that were recruiting patients. And we came upon ZOLGENSMA and, you know, I made one phone call, and they called us back that, that very night, and said “we’d like to see your child.”

PATTI: They took a working gene and sent it to replace the nonworking gene by doing an infusion that took roughly an hour. I will always remember the day when Addy received her treatment. It was at 3:42 on October 28th. I remember telling my husband, I feel like she’s having another birthday. The biggest thing that we noticed was her head control. She was able to sit unassisted for a very lengthy time.

BRANDON: You see her starting to be able to move her legs more, move her feet more, move her head. ZOLGENSMA was able to give Addy a second chance to be able to breathe freely to be able to roll herself around freely. She continues to get stronger little by little every day. To the outside eye, it may not be anything. But when you live with her every day, you see these little small things.

PATTI: We do a lot of therapy with her. She just aged out of early intervention, so prior to aging out, we did occupational and physical therapy at home as well as off site. We do water therapy with her. Again, this treatment has changed her life tremendously, but we still have a lot of underlying issues that we have to deal with that come along with having a child in a wheelchair. It’s important for people to understand that regardless of the treatment, your child has this diagnosis that’s gonna follow them for the rest of their life. Stopping the progression of SMA means not watching my child decline. It means watching her actually have the ability to feed herself. Have the ability to propel herself in her wheelchair, which is something I never thought I would see. Parents should know about ZOLGENSMA because having something that they receive one time changed our life tremendously. It’s changed Addy’s life tremendously.

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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.