SMA Atlas

Navigating life with SMA after treatment

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SMA Atlas

Navigating life with SMA after treatment

ALL TOPICS > MEMORABLE MOMENTS > Maisie says, “Momma”

Maisie says, “Momma”

A small moment that means so much

Hear from an SMA caregiver who thought she would never hear her daughter say “Momma.”
Read the transcript

CIJI:

One of my most memorable moments was, we were driving home from Utah and Maisie was in the back.

And she just started going, “Ma-Ma-Ma.”

I wasn’t sure if I was ever gonna hear that.

Without treatment, SMA [Type 1] robs their abilities to smile, to talk, to ever lift their head, to ever speak.

I couldn’t pull over immediately because I was driving on I-70 and I was like, “I gotta find a spot baby, but I hear you, I hear you.”

I pulled over and I just held her and loved her and was like, “Yay, you said ‘Momma.’”

Then when I tried to get back in the car to drive, she was like, “Ma-Ma-Ma-Ma.” And then it was like, “Hey, Maisie, I need to drive.” But the whole trip home was Ma-Ma-Ma.

It was pretty awesome.

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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

The first day of preschool was just one of the many monumental firsts for Matteo. Watch this video to see his family’s excitement and hear how much school means to them.

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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.