Public Health Genetics, Gene Therapy, and Duchenne Muscular Dystrophy

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.