Gene Therapy for Duchenne Muscular Dystrophy (DMD) Administered to 5-Year-Old Boy at Nationwide Children’s Hospital in Ohio

In a landmark moment for the Abigail Wexner Research Institute at Nationwide Children’s Hospital, a 5-year-old from Bellefontaine, Ohio, received the first dose of a recently approved gene therapy for Duchenne muscular dystrophy at Nationwide Children’s Hospital, where the therapy was invented and initially tested. The therapy--developed, tested, and patented at Nationwide Children’s--marks an extraordinary achievement in the field of neuromuscular medicine and provides a life-altering treatment for children with Duchenne muscular dystrophy. This is the second gene therapy developed at Nationwide Children’s to receive FDA approval, two of only eight to have received FDA approval to date.