Meet Kid Captain Maggie Larson
Slowing disease progression
John Bernat, MD, PhD, a clinical geneticist at UI Stead Family Children's Hospital and one of the only people in Iowa who studies the disease, told Maggie’s parents that even if she didn’t enter the clinical trial, he wanted to continue following her because of the rarity of the condition. It’s estimated between one in 40,000 to one in 160,000 people have the disease.
Under the clinical trial, Maggie receives weekly infusions of the enzyme her body lacks into the fluid surrounding her brain and spinal cord, which her parents believe have helped improve her symptoms and quality of life.
“I think our expectations have become more realistic,” Heidi adds. “We’ve seen more movement out of her. We’ve seen more communication. She smiles. She thinks it’s hilarious when her brothers get in trouble.”
And just this year, Maggie began giving the “thumbs-up” sign at appropriate times. “That’s huge,” Heidi says.
Feisty, sassy girl
Now 6 years old, Maggie is in first grade and absolutely loves school. Maggie loves being around other students and staff. She also likes “girlie” things, including Barbies and cheerleaders, and has her own pom-poms.
“She is feisty. She is sassy,” Heidi says. “Maggie communicates with her eyes. If she doesn’t like what you’re saying, she’ll repeat ‘no’ to you over and over (by closing her eyes) and finally look away. She’s hilarious.”
Her parents attribute Maggie's quality of life primarily to the care she has received at UI Stead Family Children's Hospital.
“We have found a support system that has given us hope and that has helped Maggie thrive,” Heidi says. “We cannot say enough about the care she and we have received, and, in spite of how hard it is to have a terminally ill child and know that their life is limited, we are so grateful that they are doing everything to make sure that her life is full and that she has every opportunity to be a kid.”
“She has taught us to love with reckless abandon, because we know that our time is limited,” Heidi adds, “and she’s taught a lot of other people that, too.”