Every Day With SMA

Navigating life with SMA

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Every Day With SMA

Navigating life with SMA

A day with Natalie


Learning, laughter, and lasagna

Follow Natalie through some of her favorite parts of the day and hear how her parents have balanced the different aspects of her care routine, right down to a delicious dinner.
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AARON: Celeste and I will wake up and get ourselves ready first because once your SMA child is awake, then a lot of your attention is devoted to your SMA child.

CELESTE: We put her hair up and put her shoes on and then get her ready for breakfast right away. We have to make sure that we’re giving her the nutrition that she needs to make sure muscles can keep up with her activities because it does take a little more effort for her.

CELESTE: Right after breakfast, it’s time to take her to a small in-home daycare. One of the best things that they do is physical activity with her. And then she’ll have her lunch and have a nap. It’s really important that Natalie gets a couple naps a day because we like to make sure that she gets a recharge. She needs to be able to relax her muscles.

CELESTE: After daycare, we usually go to physical therapy. Natalie usually goes to physical therapy 2 to 3 times a week. It’s almost a 1-hour session every time. It’s really important to keep building the muscles throughout her life, especially now as she’s growing.

AARON: It’s just overwhelming and brings great joy to her—and as well as us—to be able to just see her move her arms, to balance on her knees. It’s absolutely incredible.

CELESTE: One of Natalie’s favorite meals is lasagna. She loves pasta. We try to sneak in some vegetables.

CELESTE: Natalie has been able to learn to eat almost anything. We used to worry slightly about her being able to chew harder foods, like a cracker or a carrot. And now, we really are able to feed her anything that we eat. After dinner, we try to relax and play and get some more time with Natalie.

AARON: After we play for a little bit, it’s bath time. Bath time can be a little bit more difficult for a child with SMA, so it does take 2 of us.

CELESTE: After this, we’ll read a story and brush our teeth.

CELESTE: We do follow a routine and a schedule because that’s what keeps us moving throughout the day and then we also do things just for fun. There are gonna be things that are going to be exhausting and things that seem repetitive, but there’s also times when you’ll need to break the routine and do something that you normally wouldn’t do—just to make sure that you’re still enjoying life.

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Natalie, who was diagnosed with SMA Type 2, coloring with a crayon

Natalie’s new move

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.

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.

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 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.