Duchenne muscular dystrophy (DMD), commonly known as Duchenne, is a rare disorder resulting from mutations in the DMD gene, which is responsible for producing the dystrophin protein. These mutations result in insufficient production of dystrophin.
Dystrophin is crucial for preserving the structural integrity and functionality of muscle cells. The absence of functional dystrophin leads to a gradual decline in muscle strength, affecting mobility and leading to heart and respiratory issues, which significantly increase mortality rates.
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