Karson was at CHKD to receive a newly approved gene therapy for Duchenne muscular dystrophy, which he was diagnosed with at 20 months of age. Duchenne is a devastating genetic illness that causes progressive muscular wasting. Affecting mostly males, the disease causes life-threatening heart and respiratory problems as it advances. “Patients usually lose the ability to walk in their early teens and generally succumb to the illness before age 30,” said Dr. Crystal Proud, Karson’s pediatric neurologist at CHKD.
