Meet Winnie

When Winnie was born, her newborn screening tests all came back normal, and she seemed perfectly healthy. However, as she grew older, her parents noticed that she was missing certain developmental milestones, such as pulling to stand and bearing weight on her legs. Concerned, they sought medical advice. After months of testing, Winnie and her family finally got an answer. She was diagnosed with Spinal Muscular Atrophy (SMA) type II at 1-1/2 years old.

Following this diagnosis, Winnie's parents knew they needed the best care for their daughter. Luckily, they didn't have to go far. Research kept pointing them to Nationwide Children's Hospital, just three hours away from their hometown. Nationwide Children's has been dedicated to researching cures and treatments for SMA since 2010. Many research trials and breakthroughs have come from the labs of various researchers at Nationwide Children's including Dr. Jerry Mendell who pioneered another drug to treat SMA in children under 2. As soon as Winnie's parents entered the hospital, it was clear that they had made the right choice. “Immediately, the team at Nationwide Children's welcomed us with open arms,” says Ann, Winnie's mother.

One week prior to Winnie's first appointment at Nationwide Children's, the FDA approved the first-ever treatment for SMA, Spinraza. With support from her medical team, Winnie became one of the first patients to receive the drug at Nationwide Children's. “Spinraza saved Winnie's life and changed the course of her disease,” says Ann. After 11 doses, Winnie was able to stop taking the medicine.

Winnie was granted access to two additional therapies. These therapies include Evrysdi, an oral treatment for SMA which helps her body produce more of a vital protein called survival motor neuron and a weekly injection intended to create more muscle mass. Winnie is also in physical therapy.

In March of 2024, Winnie underwent surgery to correct her scoliosis, a result of her SMA. Doctors placed magnetic growing rods around her spine to correct her spinal curvature and prevent further damage. She spent five nights recovering in the hospital and was out of school for six weeks. There were no complications from the surgery and so far, it appears to have been successful in preventing further development of her scoliosis.

Today, Winnie is a spunky 9-year-old who enjoys reading, crafting, playing with her brothers, and traveling. Winnie uses a mechanical wheelchair to navigate her world but does not allow that to hold her back from doing the things she loves. Winnie is currently working on her first children's book, 'Winnie's Big Day,'' about her experience with surgery. She hopes that it will help other children feel less scared about their own surgeries.