MORGANTOWN, W.Va. – WVU Medicine Children’s continues to make bold strides in pediatric healthcare having administered the first dose of ELEVIDYS in West Virginia.
Alexander Shell
ELEVIDYS is the first approved gene therapy for Duchenne muscular dystrophy (DMD), one of the most severe forms of inherited muscular dystrophies. The therapy provides a potentially life-altering treatment for children with the disease.
The patient, six-year-old Alexander Shell, is among the first group of patients across the country to receive the gene therapy since its accelerated approval by the U.S. Food and Drug Administration (FDA) in June.
“This is truly breakthrough medicine, providing hope for Alexander and his family as well as all West Virginia families battling DMD,” Sara McGuire, M.D., pediatric neurologist at the WVU Medicine Children’s Neuroscience Center, said. “We have an amazing team here at WVU Medicine Children’s who have worked extremely hard to get this therapy approved. We are so pleased with the results.”
Alexander is also autistic and mostly non-verbal. His mom describes him as a happy child who loves tools, cars, and building things. For his family, this could be life changing.
“For us, it means he may actually be able to have some semblance of a normal childhood – not having to worry about him making a drastic change from walking to a wheelchair during the formative years of growing,” Eden Antonia Shell, Alexander’s mother, said. “For me, that means everything. He’s my only child. I want the best for him.”
Duchenne muscular dystrophy is an inherited, progressive, and fatal neuromuscular condition that impacts mostly male patients. Over time, children with DMD develop problems with walking, breathing, and heart function.
Early studies of ELEVIDYS hope to improve outcomes by slowing the progression of the disease and improving motor functions. Long-term studies to show how beneficial this treatment will be for these patients over time are still pending.
“While studies are ongoing, the hope is this medication will slow disease progression and improve outcomes, which could include extending life for these patients, whose average life expectancy is usually in their 20s or 30s,” Dr. McGuire said. “The full impact of the medication will not be known for many years but will allow the patients of West Virigina to have the option to explore these treatments for rare conditions.”
To learn more about treatment for Duchenne muscular dystrophy and the WVU Medicine Children’s Neuroscience Center, visit WVUKids.com.