Once a week, 12-year-old Ben Schussler boards a 9 a.m. plane near his hometown of Eagan, Minnesota, and flies – usually with either a parent or a “Ben’s Team” member – to the Eastern Iowa Airport in Cedar Rapids, Iowa. From there, he and his escort drive 25 minutes to the clinical research unit located within University of Iowa Hospitals & Clinics.
Clinic staff all know Ben, and he enters the clinic and heads to his infusion bay, where for an hour he’ll receive an experimental treatment for Duchenne muscular dystrophy (DMD). After the infusion, staff will monitor Ben for side effects, and on some days he has to have a blood draw to test progress. He’s usually back home by 7 or8 p.m. that same day.
Ben is part of a clinical trial testing to see whether a new therapy will change the progression of Duchenne muscular dystrophy, says Katherine Mathews, MD, pediatric neurologist at University of Iowa Stead Family Children’s Hospital and the UI’s principal investigator for the study in which Ben is participating. DMD is a genetic disorder marked by progressive degeneration of the muscles and increasing weakness.