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Navigating life with SMA after treatment

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SMA Atlas

Navigating life with SMA after treatment

Celebrating “Inchstones” in SMA

Slade, who was diagnosed with SMA Type 1, sitting with his family

Morgan and Daryn couldn’t have hoped for a happier or more beautiful little boy than the one who came into their lives in January 2019. Little did they know, the bundle of joy they named Slade was starting a journey that was going to change his and his family’s life.

About 2 weeks after Slade was born, Morgan and Daryn noticed that he was a bit floppy when holding him. Their pediatrician chalked it up to him being a calm baby and told them he’d eventually grow out of it. After a second opinion from a pediatric neurologist and 3 months of physical therapy, they were still feeling uncertain about why Slade wasn’t getting any stronger. Dissatisfied with the lack of answers, they decided to bring him to Texas Children’s Hospital for genetic screening, which eventually led to a diagnosis of spinal muscular atrophy (SMA) Type 1 when Slade was 5 months old. Even though they were scheduled for an appointment with a neuromuscular specialist a week later, Morgan and Daryn were already conducting their own research. They were determined to understand what having SMA would mean for Slade.

Through their research, Morgan and Daryn learned about 2 FDA-approved treatments for SMA. One of those treatments, ZOLGENSMA® (onasemnogene abeparvovec-xioi), had just been FDA approved 2 weeks before Slade’s diagnosis. While they waited for Slade’s infusion of ZOLGENSMA, Morgan and Daryn opted to start Slade on the other FDA-approved treatment. They felt strongly that ZOLGENSMA could help Slade and, when he was almost 7 months old, he received ZOLGENSMA. Slade also continues with the other treatment today.

Please see the Indication and Important Safety Information below and the accompanying Full Prescribing Information.

Small moments, lasting memories

Fast-forward to 6 months after his treatment with ZOLGENSMA, and Slade is now 13 months old. While he has experienced challenges as a result of his SMA, his parents are encouraged by the progress he’s made since receiving treatment. “We like to focus on Slade’s inchstones, not milestones, because every little improvement is a huge win for him and is more than we ever expected,” shares Morgan.

While simple things such as a shared look, sitting together at the dinner table, or playtime with a sibling may be taken for granted in most families, they are all precious to Slade’s parents. Since his treatment started, these inchstones have appeared throughout Slade’s daily routine. “Now, he can hold his head up and support his upper body as long as he cares to stay sitting,” says Morgan. “He’ll roll off the mat we lay him on in our living room, and we can’t leave him on the bed because he will try to scoot right off.”

Slade, who was diagnosed with SMA Type 1, with his brother

For his parents, Slade’s progress has been wonderful to watch, and with the addition of specialized medical equipment and regular therapies, his improvements are even more visible. “He seems to understand that the treatments, therapies, and equipment are all for his benefit, and he’s very tolerant of the process,” says Morgan. “He loves his medical stander and can be upright for about 30 minutes. Usually, his brother will push him up and down the hallway, which Slade loves.”

Morgan and Daryn are big believers in the importance of ongoing therapy to help manage Slade’s SMA. Speech, occupational, and physical therapy are all an integral part of Slade’s weekly care. They realize that hard work may produce only small improvements, but it’s worth it to see Slade gain strength and confidence.

Strength of spirit

Slade, who was diagnosed with SMA Type 1, sitting on a couch

While Slade has made progress physically, his parents point out that his incredible spirit also shines. “He finds joy in everything. Just the other day he was splashing around in his bath and making the most of it,” shares Morgan. Slade has also been quick to share that joy with his family by finding his voice. “Despite being speech delayed, Slade is talking more and more. He’s really starting to bloom in that area,” says Morgan.

As he grows, Slade may continue to face challenges. But with an emphasis on his progress through inchstones, his family can see his improvements more frequently. “My favorite inchstone to date is being able to sit down to dinner with Slade,” Morgan recalls. “For the longest time he had to lay flat, but now he’s strong enough to sit up at the table and be a part of what we’re doing. He’s very vocal about not being left out.”

One year out from the day Slade was diagnosed, Daryn and Morgan reflected on his SMA and shared a little about what helps them feel grateful, saying, “One thing we’ve learned is not to take a single thing for granted. Not the day, not the ability to walk, to talk, to swallow, to breathe, to scowl, and to fight with your siblings,” shares Morgan. They hope Slade will keep reaching new inchstones, and they are excited to celebrate each one together as a family.

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

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

Maisie says, “Momma”

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A photo collection of Evelyn and her sister Josephine

SMA then and now

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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 a patient experiences unexpected bleeding or bruising.

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 AveXis at 833-828-3947.

Please see the Full Prescribing Information.