Every Day With SMA

Navigating life with SMA

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Every Day With SMA

Navigating life with SMA

A day with Ryker


The right perspective

Hear Ryker's mother talk about his daily care routine with SMA and how choosing gratitude gave her a new perspective.
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STEPHANIE: I get Ryker up every morning around 7:00 or 7:30, or really just when he wakes up. And he, since he’s been having his G-tube feed overnight, I flush his G-tube with water.

We suction his nose every morning so that he doesn’t have any difficulties breathing through his nose, and then we come upstairs and we start breakfast.

Ryker can eat by mouth. He does have a safe swallow. He does have some oral motor weakness that we’re working on with his feeding specialist.

After breakfast in the mornings, depending on the day, Ryker does occupational therapy and physical therapy.

Ryker’s therapists show us what they’re doing. And they give us instruction so that we can continue those therapies at home.

So the things that we do at home for Ryker are gonna be a lot the same that we do at or in all of his different therapies.

I try to integrate all of his therapy into his normal everyday play so that it’s not different. Because all the things that he’s doing are skills and abilities that he needs every single day for daily life. So we work on stairs, we work on crawling, we work on standing up.

Ryker usually takes a nap around noon or 1:00, and during his naptime, I’m usually either cleaning up around the house or sometimes I’ll do some personal care. You know, I think it’s really important to make sure you’re getting the things done that you need to get done. But also taking the time to get some things done that you want to get done.

After Ryker wakes up from his nap he usually has a snack and then we play.

Ryker’s bedtime routine typically looks like, first we do the bath and then do his nightly breathing treatment. A full treatment, with suction and cough assist.

Cough assist machine blows a big breath of air in and then sucks it out. So it simulates a really strong cough. And he uses that so that he can, we can make sure that he’s got all of the secretions out of his lungs.

And then we read stories for a little bit. He loves books. He is a bookworm. Then we get is G-tube all hooked up to his button, and we get his pulse ox put on his toe, and then we do more snuggle time, and then we put him to bed.

There was another SMA mom that told me something that stuck with me for all of these years and that was that, “even though this life is different, it’s still beautiful.” And I have found that to be so true. I have found that this life is different. It’s not what we were expecting, but there are still so many beautiful moments and so many things that these kids teach us and so many fun memories. There’s so many things that you can do and learn and it has taught me so much. I’m so grateful for everything that I’ve learned.

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Ryker, who was diagnosed with SMA Type 1, in his wheelchair

Ryker turns 2

It's the happiest of birthdays for Ryker, who turned 2 years old! Watch a video of his parents reflecting on this joyous occasion while making lifelong memories.

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

A day with Natalie

Spend a day with Natalie as she completes physical therapy, reads with her parents, and of course, eats her favorite dinner—lasagna!

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.