Public Health Genetics, Gene Therapy, and Duchenne Muscular Dystrophy

December 18, 2023, 11:00 am – 12:30 pm ET

Duchenne Muscular Dystrophy (DMD) is a degenerative disorder of skeletal and cardiac muscle that leads to progressive loss of function and shortened lifespan.  DMD is one of many rare diseases, which together affect about 25 million people in the United States and about 400 million people worldwide. While individual treatments need to be tailored to each condition, the public health considerations in achieving health equity for one condition can inform future efforts for other rare diseases. This webinar will review the population health impact of rare diseases such as DMD. Presenters will cover the genetics and clinical impact of DMD, the evolution of gene therapy, the development of FDA-approved gene therapy to treat the underlying protein deficiency that causes DMD, and the health equity challenges.

headshot of Catharine Riley

Catharine Riley, MPH, PHD,
National Center on Birth Defects and Developmental Disabilities,
Centers For Disease Control and Prevention,
Atlanta, Georgia

headshot of Seth Perlman

Seth Perlman, MD,
Seattle Children’s Hospital & University of Washington,
Seattle, Washington

headshot of Russell Butterfield

Russell Butterfield, MD, PHD,
University of Utah Health,
Salt Lake City, Utah

headshot of Joon-Ho Yu

Joon-Ho Yu, MPH, PHD,
University of Washington,
Seattle, Washington

Hosted by

  • Public Health Genomics Branch and Rare Disorders and Health Outcome Team, Centers for Disease Control and Prevention