Synopsis of Program Content:
Within the last four decades, the evolving natural history of Duchenne Muscular Dystrophy (DMD) continues to affect both survival and the loss of clinically meaningful motor functions. Various medical interventions such as the efficacious use of corticosteroids and genetic alteration of the dystrophin gene have impacted the insidious course of this disease’s progression and quality-of-life issues by measurably prolonging the duration of muscular capacity.
In recent years, advances in medical research and a better understanding of the pathophysiology of DMD have allowed the development of novel agents and improved treatment strategies for patients that were left previously with minimal therapeutic options. This CME activity will examine the typical and ameliorated natural history of DMD, instruments for surveillance of clinical course, and administration of medicines with proven efficacy for individual patients. Equally important, expert faculty will address an explanation of the data necessary to prove medical necessity to health insurance providers and empower families to work with insurers.
After completing this CME activity, the participant will be able to:
1. Discuss the natural history of DMD and the factors that can facilitate early diagnosis and treatment.
2. Evaluate the prevailing evidence surrounding how genetic testing can inform the direction of clinical research in treating neuromuscular degeneration.
3. Recognize important criteria documented in the 2016 AAN evidence-based guidelines for the management of DMD using currently available treatment options.
4. Contrast current and emerging therapies for DMD to demonstrate how different therapies can be used to individualize patient care.
5. Discuss endpoints for measuring clinical course in patients with DMD and the challenges of demonstrating necessity of treatment for reimbursement.
Perry B. Shieh, MD, PhD
Assistant Professor of Neurology
Director, Neuromuscular Program
David Geffen School of Medicine at UCLA
Los Angeles, CA
John W. Day, MD, PhD
Professor of Neurology and Pediatrics
Director, Division of Neuromuscular Medicine
Stanford University School of Medicine