Every Day With SMA

Navigating life with SMA

Scroll Down Icon

Every Day With SMA

Navigating life with SMA

Natalie’s new move


A family win

The times when children are proud of themselves are heartwarming moments for parents. Watch Natalie’s parents talk about one such moment and the impact it had on their family.
Read the transcript

CELESTE: One of the most meaningful moments after treatment was when Natalie was able to stand for a short period of time with support.

AARON: She’s still not able to walk or stand by herself, but when somebody is able to kind of hold her up just around her chest and stand for at least a few seconds—to see just her standing vertically instead of just rolling on the floor, it just warms my heart to see that.

CELESTE: She was standing and kept looking down at her feet and then looking back up. And then as soon as her Dad looked at her, she said, “Dad, dad, see?” And she was so excited to show off her new move.

CELESTE: That meant so much to us because not only did we know that it was a big deal for her, but she herself knew that she was doing something that she hadn’t been able to do before.

AARON: She knew that she had hit a milestone. And it was just absolutely incredible to see the happiness on her face and she knew that she was going places.

Close the transcript
Donovan, who was diagnosed with SMA Type 1, playing with his dad

Preparing for the future

Hear the perspectives of multiple families about why it’s important to be knowledgeable about SMA and its management even after treatment.

Matteo, who was diagnosed with SMA Type 1, walking with his mom and dad

Matteo’s first day of school

Hear an SMA family describe the moment their son walked into class on the first day of school and why it means so much to them.

Preparing for the future

Check out resources and advice from other caregivers to help you navigate your child’s future with SMA.

Get started

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.