SMA Atlas

Navigating life with SMA after treatment

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SMA Atlas

Navigating life with SMA after treatment

Adventures abroad with our child with SMA

Evelyn, who was diagnosed with SMA Type 1, traveling with her mom and dad

Traveling is a wonderful way to learn about other cultures, find time to relax, and bond with your family while making lifelong memories. And while any trip requires careful planning, traveling with a child who has spinal muscular atrophy (SMA) comes with added considerations and difficulties. Evelyn’s parents worried that her SMA diagnosis might limit their ability to travel and gain those valuable experiences. However, Evelyn’s family has seen her progress nicely and gain confidence since receiving treatment and felt ready to embark on their first trip abroad. With a plan in place and passports in hand, Evelyn and her family set their sights on Switzerland. Not only was it a major milestone for the whole family, but it was also fun and exciting. Read on to hear from Evelyn’s parents, Milan and Elena, about their experience.

Results and outcomes vary among children based on several factors, including how far their SMA symptoms progressed prior to receiving treatment.

How did you get Evelyn excited about the trip?

We told her we’re going on an airplane. She was super excited about that part and kept telling anyone who would listen about the nap she was going to take on the long plane ride. But also, we learned about Switzerland as a country. We looked at a map and showed Evelyn that we were going across the ocean. We talked about the mountains and fun facts about Switzerland. And of course, Evelyn was a trooper on the flight, but she didn’t sleep until we arrived and started walking around. We put her in the stroller, and she went out like a light.

How did you prepare to travel?

We created this folder about specific things to do and see in Switzerland and planned ahead the places we were going to stop. We wanted to live like locals while we were there, so we stayed in an Airbnb and went grocery shopping so we could cook our meals at home. We did our best to design the trip to be budget-friendly so we could enjoy doing more. Calling ahead to make sure the activities we wanted to do were family-friendly and manageable for Evelyn was a big part of our preparation. Evelyn has weakness in her legs and gets tired easily from walking, so we made sure to consider her limitations when picking our daily activities.

What was her favorite part of your trip?

Evelyn loved the gondolas. One day we took a ride on one into the mountains to do a family hike. When the terrain was too difficult for Evelyn, she rode on our shoulders. Once we got into the flat areas, she was able to walk and play. We were just a family together up in the mountains, and it was great to see Evelyn play.

What did this trip mean to you as a family?

It was such an incredible trip. From beginning to end, it was wonderful. We got to be together as a family and do something we never expected to do after Evelyn’s SMA diagnosis. We love being world travelers and want to teach her to enjoy the world as much as possible. It was a great chance to learn what we can accomplish together.

The next adventure

Evelyn definitely caught the “travel bug” and enjoyed their trip so much that she can’t stop talking about all of her favorite parts. Although her family still finds time for short day trips to the beach, her parents are pressing pause on another long trip until their new baby daughter, Mila, gets a little older. For Evelyn, the next mountain to conquer can’t come soon enough.

See more about Evelyn’s experience with ZOLGENSMA and what she is up to as a 4½-year-old. Every child with SMA has different needs and may not be like Evelyn. Check out these helpful tips when traveling with a child with SMA. Talk with your doctor to determine your child’s abilities before traveling.

Maisie, who was diagnosed with SMA Type 1, saying, “Momma”

Maisie says, “Momma”

Hear about Maisie’s journey to treatment and the joy her mother felt when she heard Maisie say “Momma” for the first time.

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.

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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.