Pharmacovigilance and Risk Management Strategies Conference

Rare diseases present special challenges for patients, caregivers, and those who seek to develop and provide safe and effective treatments. One disease that presents such challenges is Duchenne Muscular Dystrophy (DMD), a genetic disorder that is characterized by progressive muscle degeneration and weakness caused by an abnormality in the dystrophin complex in muscle fiber. Development of safe and effective biopharmaceutical interventions has been enigmatic. This session will provide an overview of this devastating disease, patient advocacy efforts, and the partnership network that has emerged to facilitate development of evidence-based interventions.