HJAR Jan/Feb 2025

36 JAN / FEB 2025 I  HEALTHCARE JOURNAL OF ARKANSAS ADH CORNER COLUMN CHILDREN’S HEALTH Duchenne muscular dystrophy (DMD) is the most common muscular dystrophy in children, affecting boys. While this devastating, progressive neuromuscular disorder has no cure, several gene therapy programs are making strides to improve patient outcomes. I serve as the principal investigator on five current DMD gene therapy trials. In the past six years, I have been the principal investiga- tor for over 15 Duchenne clinical trials, treat- ing over 100 patients living with Duchenne and Becker muscular dystrophy. I want to ensure that children from Ar- kansas and neighboring rural states have equal opportunity to access these clinical trials. Historically, if you look at clinical trial participation, representation from the un- derserved rural states such as Arkansas is minimal. An estimated 15,000 boys in the U.S. live with DMD and about 300,000 worldwide. It causes muscle weakness due to a mutation in the dystrophin gene. Duchenne is a mul- tisystem disease affecting skeletal muscle, cardiac muscle, respiratory muscle, learning, and behavior. Boys lose ambulation around 11 to 12 years and develop respiratory dys- function and cardiomyopathy in their teens. Currently, there is no cure for this disease. Though available treatments such as cor- ticosteroids and newer treatments such as exon-skipping therapies can slow down the Duchenne Muscular Dystrophy Gene Therapy Changing Trajectory of Disease, Lives