Every Day With SMA

Navigating life with SMA

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Every Day With SMA

Navigating life with SMA

Daily life

You will encounter new opportunities and different challenges every day when living with spinal muscular atrophy (SMA), even after treatment. Check out these tips and ideas from caregivers to help you get through each day with more confidence.

Ryker, who was diagnosed with SMA Type 1, laughing while looking in a mirror

A day with Ryker

Hear about the daily routine for Ryker, who was diagnosed with SMA Type 1, and the philosophy of gratitude that his mother has embraced.

Slade, who was diagnosed with SMA Type 1, laying on a blanket and playing with a truck

A day with Slade

When Slade was diagnosed with SMA, his parents knew they would have to find a care routine that worked for their family. Hear how his parents stick to a schedule to set Slade up for success.

Natalie, who was diagnosed with SMA Type 2, sitting in a swing

A day with Natalie

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.

A paper family holding hands

Fun for the whole family

For a child with SMA, finding activities the whole family enjoys can sometimes be challenging. Check out these caregiver-suggested fun family activities for in and out of the home.

Ryker, who was diagnosed with SMA Type 1, laughing in his dad's arms

Staying connected while staying home

Families living with SMA need to isolate when flu season hits or when an illness is affecting the community. Hear from caregivers about the challenges of staying home and their advice on staying connected with others.

Icon of camera

Celebrate memorable moments

From a birthday party to standing up with a little help, check out special moments that children are experiencing.

Watch the videos

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.