Updates in Duchenne’s Muscular Dystrophy: A Focus on Emerging Gene Therapy

TARGET AUDIENCE:
This activity is intended for pediatric neurologists, neuromuscular specialists, developmental neurologists, hospital administrators, and clinical care staff. The secondary target audience is general neurologists, pediatricians, geneticists, genetic counselors, physical therapists, and other clinical staff involved in the multidisciplinary management of patients with DMD.

PROGRAM OVERVIEW:
Newborn screening, early diagnosis, and genetic testing of known carriers for DMD are not happening. It is critically important to recognize and diagnose DMD as early as possible, because earlier treatment has the potential to double the lifespan.Clinical trials for gene therapy in DMD are starting to show encouraging results. The rapid advancement of the field dictates continued education of patients, HCPs, payors, and policymakers on the progress that has been made since early trials. This program will explain the genetic causes of DMD, the natural history of the disease, and how gene therapies may be used to treat DMD. Data from three clinical trial studies are reviewed.

LEARNING OBJECTIVES:
Upon completion of the activity, participants should be able to:
• Review the genetic underpinnings of DMD
• Describe the association of gene transfer therapy components: its promoter, transgene and vectors, the outcomes of expression, function, and safety, respectively
• Compare expression, function, and safety outcomes data for the gene therapies presently under investigation for DMD
• Evaluate optimal approach to the assessment of gene therapy, related clinical effects, safety, and efficacy