INDIANAPOLIS — Officials with the Riley Hospital for Children recently announced that the hospital is the first one in Indiana to administer gene infusion therapy for Duchenne muscular dystrophy.
A 5-year-old northern Indiana resident was the first recipient of the treatment on Oct. 23, a release from the hospital said. Officials said that Duchenne muscular dystrophy is a severe, life-limiting genetic disorder that can lead individuals to a decline of mobility, independence and an overall quality of life.
“We are honored to be the first children’s hospital in Indiana to provide gene infusion therapy for Duchenne muscular dystrophy,” Riley Children’s Health Chief Medical Officer Dr. Mara Nitu said in the release. “Our mission has always been to provide the best possible care to the patients and families we serve. Being able to offer this therapy underscores our commitment to giving our patients a chance at a brighter future.”
Officials said that this therapy is a “ray of hope” for the children affected with Duchenne muscular dystrophy, as well as their families.
“In clinical trials it improved both the amount of dystrophin protein in their muscles and their motor function, while untreated children declined,” Dr. Larry Walsh, a pediatric neurologist with Riley Children’s Hospital, said in the release. “The long-term outlook is still unknown, but anything we can do to preserve or improve function in children affected with DMD gives us reason for optimism.”