Duchenne muscular dystrophy (DMD) is a rare, genetic muscle disease that primarily affects males and is characterized by muscle loss that worsens continuously as boys age. Loss of strength and function typically first appears in pre-school-age boys. As the disease progresses, the severity of damage to skeletal and cardiac muscles results in patients experiencing a total loss of ambulation in the pre-teenage or early teenage years. Disease progression in the teen years typically includes worsening cardiac and respiratory symptoms and loss of upper body function. There are no transformative therapies and no cure for DMD.
Living with DMD has profound impacts on every part of people’s lives, from work and socializing to family life. Community members have shared moving examples of living with Duchenne with our team and have told us what it would mean to them to have a transformative therapy for their disease.
Every year on September 7th, we join the Duchenne community in recognizing World Duchenne Awareness Day.
DMD is the most common childhood-onset form of muscular dystrophy. Symptoms are often first detected between 3 and 5 years of age.
DMD results from a genetic mutation in the DMD gene on the X chromosome. This gene regulates the production of dystrophin, a protein essential to healthy muscle development and function. In people with DMD, dystrophin levels are absent or nearly absent, which causes permanent damage to muscle cells. DMD primarily affects males. Females with a DMD mutation can be affected. They also pass the genetic mutation on to their children.
People living with DMD experience a range of symptoms, which may include:
There is a significant need for new treatment options for DMD.
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