Duchenne muscular dystrophy (DMD) is a genetic condition in which an individual lacks a functional version of the dystrophin gene, which helps muscles stay healthy. As a result, their muscles break down and are, over time, replaced with fatty deposits. This leads to a host of symptoms, from loss of mobility to life-threatening heart and respiratory problems to low life expectancy.
While there is no known cure for DMD, experts in the Neuromuscular Program at Children's Hospital of Philadelphia (CHOP) are active participants in clinical trials, working to create new and innovative treatments for neuromuscular disorders. For these efforts, the program was recently named a Certified Duchenne Care Center (CDCC) by the Parent Project Muscular Dystrophy (PPMD). This certification is given to programs that maintain the highest standards in clinical and sub-specialty services, rapidly apply new evidence-based knowledge, and comply with CDC clinical care standards. CHOP is also the first center to receive the PPMD’s newly established Clinical Research Designation — recognizing excellence in both clinical care and research. This certification highlights CHOP’s long-standing commitment to providing world-class care and advancing clinical research for individuals living with muscular dystrophy.
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Duchenne muscular dystrophy (DMD) is a genetic condition in which an individual lacks a functional version of the dystrophin gene, which helps muscles stay healthy. As a result, their muscles break down and are, over time, replaced with fatty deposits. This leads to a host of symptoms, from loss of mobility to life-threatening heart and respiratory problems to low life expectancy.
While there is no known cure for DMD, experts in the Neuromuscular Program at Children's Hospital of Philadelphia (CHOP) are active participants in clinical trials, working to create new and innovative treatments for neuromuscular disorders. For these efforts, the program was recently named a Certified Duchenne Care Center (CDCC) by the Parent Project Muscular Dystrophy (PPMD). This certification is given to programs that maintain the highest standards in clinical and sub-specialty services, rapidly apply new evidence-based knowledge, and comply with CDC clinical care standards. CHOP is also the first center to receive the PPMD’s newly established Clinical Research Designation — recognizing excellence in both clinical care and research. This certification highlights CHOP’s long-standing commitment to providing world-class care and advancing clinical research for individuals living with muscular dystrophy.

Gene Therapy for Duchenne Muscular Dystrophy: Cash’s Story
Children’s Hospital of Philadelphia now offers the first-ever gene therapy treatment for Duchenne Muscular Dystrophy (DMD) to patients like 6-year-old Cash. This breakthrough treatment slows the progression of symptoms and can improve strength and endurance for patients with DMD.
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Gene Therapy for DMD: Cash's Story

CHOP now offers the first-ever gene therapy treatment for DMD to patients like 6-year-old Cash.
Muscular Dystrophy
Muscular dystrophy causes the muscles in the body to become weak. The muscles break down and are replaced with fatty deposits over time.
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Neuromuscular Program