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What's New in Duchenne Muscular Dystrophy (DMD)

Activity Snapshot

Activity Type: 
Release Date: 
Wednesday, June 1, 2022


Emma Ciafaloni, MD, FAAN
Professor of Neurology, Pediatrics and Obstetrics and Gynecology, Director Pediatric Neuromuscular Medicine
University of Rochester
Activity Overview: 

This speaker slide set will provide an overview of Duchenne muscular dystrophy (DMD) and the current treatment landscape. Some topics included: clinical presentation and pathology, diagnostic considerations and current and emerging therapies.

Learning Objectives: 
  • Recognize signs/ symptoms of DMD
  • Identify ways to reduce diagnostic delay (eg, creatinine kinase, genetic testing, newborn screening)
  • Implement current treatment guidelines to manage DMD and related comorbidities
  • Use of multidisciplinary care to optimize outcomes (cardiac, pulmonary)
  • Mental health challenges, ways to reduce emotional toll of DMD
  • Identify emerging treatment strategies and ongoing clinical research (eg, gene-targeted therapy, exon-skipping) Advise patients of clinical trial opportunities