Purpose of review: This article reviewed the most recent clinical trials investigating potential treatments for Duchenne muscular dystrophy (DMD). In the development of these studies, investigators have encountered unexpected challenges that reveal limitations in our understanding of the clinical trial design for this disease.
Recent findings: Recent clinical trials for DMD have revealed limitations in our understanding of the natural history of this disease. As a result of these revelations, there has been a significant effort to document the natural history of Duchenne and develop valid outcome measure that could be used in clinical trials in different subsets of patients.
Summary: Treatment for DMD is a rapidly changing field. Some of the drugs that are currently under investigation will receive Food and Drug Administration approval in the near future. These would become part of clinical practice. This article summarizes the different therapeutic approaches under investigation and illustrates the importance of defining sound research methods that are specific to the disease and population under investigation.