Kid Captain Lily Lee receives cutting-edge treatment at Stead Family Children’s Hospital
Fort Dodge girl diagnosed with spinal muscular atrophy
Lily Lee was meeting all of her early milestones, but her parents became concerned when she still wasn’t walking well after her first birthday.
“It was a completely perfect normal pregnancy and birth,” says Lily’s mother, Mya. “There was not a single issue.”
When Lily still wasn’t walking at 14 months, Mya contacted their local pediatrician. At first, the doctor wasn’t overly concerned. But over the next two months, Lily began to progressively weaken. By 16 months, she could take only a few unsteady steps. Tests at a local hospital didn’t reveal anything abnormal, so the family was referred to University of Iowa Health Care Stead Family Children’s Hospital.
Diagnosis at Stead Family Children’s Hospital
Lily started genetic testing when she was 20 months old. She was examined by pediatric neurologist Seth Perlman, MD, who suspected the toddler had spinal muscular atrophy (SMA), an often fatal neuromuscular disorder that causes muscle deterioration and weakness.
Several weeks passed before Dr. Perlman’s suspicions were confirmed.
Mya remembers sitting in her car crying after hearing Lily’s diagnosis in early 2016. At the time, no treatment was available and most children died of the disorder before their teens.
“We were devastated to learn that our perfectly healthy daughter’s muscles would slowly deteriorate over time and that nothing could stop the progression of the disease. There was absolutely nothing we could do to help her,” Mya says, adding that Perlman offered compassion during an incredibly difficult period. “He took the time to explain everything with care and made us feel supported and not alone. His kindness and understanding helped us face an overwhelming diagnosis with hope and trust.”
New therapy approved
Just months after Lily’s diagnosis, news came that would change everything. Shortly before Christmas, Mya received an email: The FDA had approved Spinraza, the first treatment for spinal muscular atrophy. Designed to slow the progression of SMA and improve muscle strength, the drug offered something the family had been missing since Lily’s diagnosis: hope.
Hospital staff helped obtain insurance approval and by February 2017, Lily received her first shot, the first patient at Stead Family Children’s Hospital to be treated with the drug.
She now receives treatment through a lumbar puncture in her spine every four months to stop progression of the disease. Lily also participates in a clinical research study that requires monthly infusions, intended to help build muscle.
At about the same time she started genetic testing, Lily received her first wheelchair.
“The second that she sat in this little, tiny wheelchair, she went all over the place,” Mya recalls. “At this point, she could crawl, but if she went anywhere, she was just being carried by us. But with this wheelchair, she just started going. She just knew how to do it.”
Another memorable occasion happened when Lily returned for one of her regular lumbar punctures, which she receives without sedation. Knowing that Frozen was her favorite movie at the time, nurses decorated the procedure room with sparkling snowflakes and other themed decorations.
“They even gave Lily an Elsa dress,” Mya says. “I remember her eyes lit up. She loved it. Small acts like this made a frightening experience feel safe and special for her.”
Child Life specialists also have helped Lily navigate the procedures, as she regularly makes the three-hour trip to Iowa City from the family’s home in Fort Dodge.
“They always make her feel special,” Mya says. “She never leaves in a bad mood. They all know her; they all love her. It’s so amazing knowing that we walk in and everyone’s happy to see her.”
Future outlook
Now 12 and in seventh grade, Lily is a good student, enjoys using her handcycle bike, and spending time with her friends at school. She loves swimming at summer camp and hopes to be a cosmetologist someday.
Her father, Chad, made parallel bars for Lily so she could practice walking. Mya says Lily’s arm strength has increased and she is able to use her arms to help her bear weight in her legs.
“When Lily was diagnosed, there was nothing,” she says, adding she is grateful the therapy became available when it did. “The sooner you get (treatment), the better off you are.”
Mya says Lily will need the treatment for the rest of her life and appreciates the care her daughter receives at Stead Family Children’s Hospital. "They continually remind Lily how brave she is and make her feel proud of herself,” she says. “Their compassion, dedication, and commitment to both her medical care and emotional well-being have meant everything to our family. I would not want her to be anywhere else.”
Kid Captain Hall of Fame
Each football season, we welcome 12 unforgettable patients into our Kid Captain Hall of Fame. Explore our four most recent stories.
Born in Michigan with a rare heart defect, Beau Harvey underwent three open-heart surgeries before his health journey led to a heart transplant at University of Iowa Health Care Stead Family Children’s Hospital, the only hospital in Iowa that performs pediatric heart transplants.
At age 4, unusual bruising led Charlie and his family to Stead Family Children’s Hospital, where he was diagnosed with severe aplastic anemia, a rare and life-threatening bone marrow disorder.
When unusual bruising appeared on Charlie Price’s back and arm at age 4, his parents began searching for answers, ultimately leading to a rare diagnosis of severe aplastic anemia.
Millie was transferred to Stead Family Children’s Hospital shortly after birth due to breathing issues, eventually undergoing open heart surgery at just 4 months old. She was diagnosed with a rare syndrome, Bannayan–Riley–Ruvalcaba.
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