Duchenne Muscular Dystrophy Clinic

The UR Duchenne Muscular Dystrophy Clinic mission is to provide evidence based multidisciplinary clinical care that comply with standards in clinical care established by the Centers for Diseases Control and Prevention to patients with Duchenne and Becker Muscular Dystrophy (DBMD) of all ages.

Our goal is to make a positive impact on the quality of life of children, adolescents and adults affected by DBMD by providing a team of experts in all specialties needed to best care for DBMD, a friendly and supportive atmosphere, and coordinated care that is highly accessible for families. We focus on all aspects of disease including a timely diagnosis with genetic confirmation, genetic and family counseling, prevention and management of secondary symptoms and complications, including cardiac, respiratory, behavioral, endocrine and bone health, access to clinical trials, and coordination of care with schools and other providers.

Teddy’s journey through experimental gene therapy for Duchenne: Dr. Emma Ciafaloni, Director of the Duchenne Clinic, and Principal Investigator for the trial, explains how gene therapy works and talks about the Department of Neurology longstanding commitment to find new treatments for Duchenne.