ACTSI-supported Clinical Trial now FDA-approved to treat Muscular Dystrophy

ACTSI’s Pediatric Clinical Research Unit at Children’s Healthcare of Atlanta, Egleston plays host to Addison (Blue) Jones, a fourth-grader with duchenne muscular dystrophy (DMD), the most common form of the disease among children and especially boys. From an early age, his family noticed his struggle with everyday activities. Two years ago, they found hope. Blue was selected for a clinical trial for Eteplirsen, a drug recently named by the FDA as treatment for muscular dystrophy. The expanding availability of this drug in hospitals across the nation will give DMD children a chance at a better quality of life.

This research, performed by ACTSI-supported researcher Han Phan, MD, assistant professor of neurology, Department of Pediatrics, Emory University School of Medicine, successfully slowed the disease’s progression, allowing children to get stronger and walk a little longer.