Every Day With SMA

Navigating life with SMA

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Every Day With SMA

Navigating life with SMA

Preparing for the future

Getting treatment is just the first step

SMA caregivers share the importance of being knowledgeable about what a child may need to grow and thrive after treatment.
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MILAN: Caregivers need to be knowledgeable about SMA because it’s going to affect the way the child grows up.

ELENA: We need to always look for things that might come up and how, how can we help these kids the best way possible. So when they grow up, it will make a big difference for their life.

TINA: We’re always looking at new therapies to see what we can do to help with Malachi gain strength. We’re going along and learning things as he grows. There’s a lot of information out there, so sometimes you just have to take a step back and kind of go and look at it at your own pace.

MATT: We’re learning now to help us prepare for the future. I think some of the advice I would say is don’t lose sight of what’s important. And not in a cliché way, but in a very real way. There’s a lot of little things that therapists tell you to do in between appointments. Take the little things seriously.

LAURA: 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 they need to grow and thrive.

TINA: This is a life that none of us anticipated. It’s nothing that we ever expected. But you will do what you need to for your child at the end of the day. You won’t let them give up, so you don’t give up.

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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.

Malachi, who was diagnosed with SMA Type 1, in a gait trainer

Advocacy doesn’t stop after treatment

Even though a child receives treatment for his or her SMA, it doesn’t mean their care stops. Hear from SMA caregivers about how they learned to be strong advocates for their children in the years following treatment.

Making progress in SMA

Learn how newborn screening and having SMA treatments can help improve the lives of children with SMA.

Hear from SMA families

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.