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Making a Difference in Duchenne Muscular Dystrophy & Spinal Muscular Atrophy: Pediatricians at the Front Line of Early Diagnosis & Improved Survival

Format
: Live Program
Topic
:
Resources
Credits
: 1.5 AMA PRA Category 1 Credit(s)™
Target Audience
: Health care providers who care for patients with DMD and SMA
Expiration Date
: 11/05/2018

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Please join us for an Industry-Sponsored Symposium at AAP 2018
Making a Difference in Duchenne Muscular Dystrophy & Spinal Muscular Atrophy: Pediatricians at the Front Line of Early Diagnosis & Improved Survival
This activity is supported by an independent educational grant from Sarepta Therapeutics

Monday, November 5, 2018
Dinner: 7:00 pm – 7:30 pm
Scientific Program: 7:30 pm – 9:00 pm

International Ballroom H, Convention Level (One Floor Above Lobby Level)
Hyatt Regency Orlando
9801 International Drive
Orlando, FL

Activity Overview

Pediatricians are uniquely positioned to provide family-centered care in Duchenne Muscular Dystrophy (DMD) and Spinal Muscular Atrophy (SMA) based on the understanding that the family is the child’s primary source of strength and support. Pediatricians need to regularly screen, identify and evaluate babies and young children for delay in neuromotor development, which is a cardinal feature of DMD and SMA. For both diseases, there is significant delay from the time of earliest symptoms to a definitive diagnosis. With current therapies that modify disease, it is critical to reduce this diagnostic delay. This CME-certified activity will review published guidelines for the early screening and identification of infants with motor delay, initial steps in evaluation and management, and recommendations for specialty referral.

For patients with DMD, intervening at the earliest stages of disease, before muscle loss and fibrosis, is critical for long-term disease amelioration. This CME-certified activity will review the utility of CK levels, and the role of diagnostic tests (such as genetic testing and muscle biopsy). We will discuss the physiologic basis and data to support current and emerging pharmacologic approaches for modifying disease progression in DMD.

Similarly, early treatment of patients with SMA, prior to the irreversible loss of motor neurons in the spinal cord and brainstem, is critical for altering disease course, preventing morbidity and mortality. This CME-certified activity will provide guidance for early recognition of SMA and review the diagnostic evaluation for children suspected of having SMA. We will discuss the pathological basis of the disease and the current and emerging approaches to treatment.

Educational Objectives

Upon completion of this activity, participants should be able to:

1.  Examine the epidemiology, presenting symptoms, and diagnostic tests for DMD and SMA, which can facilitate the early diagnosis and treatment of these diseases
2.  Be familiar with published diagnostic criteria and treatment guidelines for the management of DMD and SMA
3.  Explain the current understanding of the cellular pathophysiology of DMD and how scientific advances in the molecular mechanisms involved has led to better treatment of neuromuscular degeneration
4.  Understand the current and emerging treatment landscape for both DMD and SMA

Faculty

Katherine D. Mathews, MD
Program Chair
Professor, Departments of Pediatrics and Neurology
Carver College of Medicine of University of Iowa
Vice Chair for Clinical Investigation
Director, Muscular Dystrophy Clinic
Iowa City, IA

Richard S. Finkel, MD
Professor, Department of Neurology
University of Central Florida College of Medicine
Division Chief, Division of Neurology, Department of Pediatrics
Nemours Children’s Health System
Orlando, FL

Craig M. McDonald, MD
Professor of Clinical Physical Medicine and Rehabilitation
Professor of Clinical Pediatrics
University of California Davis, School of Medicine
Medical Director of the Spina Bifida Program
Shriners Hospitals for Children—Northern California
Sacramento, CA