Wheels, Wings and Little Wins: Traveling with SMA
Even under perfect circumstances, flying with kids can be tough—especially for the first time.
So, mom Samantha Lackey planned extensively and hoped for no surprises. She researched airline accommodations, including seating, bathrooms and wheelchair storage, and how to get through TSA with her daughter’s medication.
Of course, there were certain things Samantha couldn’t anticipate—like how her four-year-old would react to turbulence. Luckily, little Stella decided quickly: she is a big fan.
“I’m shocked at how well she did on the airplane,” said Samantha. “I was holding on for dear life! And Stella was laughing, saying, ‘It tickles my tummy!’ and ‘This is so fun!’ She’s a daredevil.”
Stella even left the plane with a pair of pilot’s wings to celebrate her bravery.
Thankfully, Stella tends to meet challenges with curiosity rather than trepidation—perhaps because she’s been defying people’s expectations since she was just weeks old, when she was diagnosed with spinal muscular atrophy, or SMA.
SMA leads to progressive muscle weakness, paralysis, and when left untreated in its most severe forms, permanent ventilation or death in 90% of cases by age two. It’s caused by a missing or nonfunctioning survival motor neuron 1 (SMN1) gene, which produces SMN protein, responsible for muscle growth and development.
Stella was diagnosed with SMA Type 1, one of the most severe forms, in 2019.
Immediately, Samantha and her husband Jeremy worked with their neurologist to seek treatment for Stella and decided on Zolgensma® (onasemnogene abeparvovec-xioi), which had just received FDA approval.
Zolgensma is the only gene therapy for SMA, and the only SMA treatment designed to address the genetic root cause of the disease by replacing the function of the missing or non-working SMN1 gene with a single, one-time dose.
“I’ve been working with patients with SMA and their families for a long time. When I was in the clinic more than a decade ago, it was heartbreaking to tell parents that there was nothing more I could do for their child’s diagnosis,” said Dr. Sandy Reyna, Global Head of Medical Engagement & Chief Scientific Advisor at Novartis Gene Therapies. “Recent treatment innovations have totally changed what life can look like for people with SMA, especially when they’re diagnosed and treated early. Today, caregivers can choose from three FDA approved treatment options.”
Zolgensma can increase liver enzyme levels and cause acute serious liver injury or acute liver failure which could result in death. In clinical trials, the most common side effects were elevated liver enzymes and vomiting. Please see additional Important Safety Information below and accompanying Full Prescribing Information. Children treated with Zolgensma need to receive an oral corticosteroid starting the day before infusion, and then after infusion for about two months or longer depending on their liver function exams and labs. Children treated with Zolgensma also need baseline labs and then need to return for blood tests weekly, bi-weekly and then monthly for at least the first three months after treatment.
“We’re seeing kids whose lives are full of possibilities, kids that are doing new things every day,” explained Dr. Reyna.
Stella was treated with Zolgensma at two months old and started an additional treatment last winter. Now, at just four years old, she is taking on new adventures every day.
“We’ve been working on independence,” explained Samantha. “At occupational therapy, we’ve been practicing transferring from her wheelchair, and at home, we’re encouraging her to do things herself. Letting her pick her toothpaste, her pajamas, giving her the opportunities to make choices and be empowered. I know that people sometimes don’t see those as big milestones, but for her, they’re huge.”
Rising to the challenge has become a part of Stella’s everyday routine.
“It’s been amazing to see how her confidence and personality has grown during this trip,” said Samantha. “When we were waiting for our return flight, she went up to a customer service desk by herself, and asked the representative if her stuffed mermaid needed a plane ticket. She didn’t even bother asking me!”
Her biggest advice for families who are traveling? If you need something, say so—just like Stella did for her mermaid.
“The most helpful tip is to not be afraid to seek out help or clarification,” recalled Samantha. “Asking for guidance definitely made our trip a lot easier.”
Samantha helped Stella ask questions about what the TSA agents were doing as they wiped down her wheelchair and looked through her medication cooler.
“It’s hard for anyone to ask for help,” explained Samantha, “but as Stella gets to be school-aged, to set her up for success, we want her to be comfortable saying, “Hey, don’t touch my wheels,” or “Can you help me?”
With one family trip down, Samantha sees more travel on the horizon. She hopes that, in the near future, Stella and other wheelchair users will be able to stay in their wheelchairs for entire flights. Until then, Samantha will continue to advocate for more accessible travel, and Stella will keep making daily progress.
“It’s been really exciting to see her little wins every day. We know how truly lucky we are for Stella to be doing the things she’s doing, and to have the support that she needs from so many people.”
Results and outcomes vary among children based on several factors, including how far their SMA symptoms have progressed prior to receiving treatment.
Indication and Important Safety Information for ZOLGENSMA® (onasemnogene abeparvovec-xioi) suspension, for intravenous infusion:
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.
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 which could result in death.
- 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. Caregivers and close contacts with the patient should follow infection prevention procedures. Contact the patient’s doctor immediately if the patient experiences 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 generally occur within the first two weeks after ZOLGENSMA infusion. 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 influenza and respiratory syncytial virus (RSV) is recommended and vaccination status should be up-to-date prior to ZOLGENSMA administration. Please consult the patient’s doctor.
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 patient body waste for one 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.
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 http://www.fda.gov/medwatch, or Novartis Gene Therapies, Inc. at 833-828-3947.
Please see the Full Prescribing Information.
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