Uncovering the Embryonic Origins of Duchenne Muscular Dystrophy
Philip Barrett,
Ke'ale W. Louie,
Jean‐Baptiste Dupont
et al.
Abstract:Duchenne muscular dystrophy (DMD) is a severe degenerative muscle disease caused by mutations in the DMD gene, which encodes dystrophin. Despite its initial description in the late 19th century by French neurologist Guillaume Duchenne de Boulogne, and identification of causal DMD genetic mutations in the 1980s, therapeutics remain challenging. The current standard of care is corticosteroid treatment, which delays the progression of muscle dysfunction but is associated with significant adverse effects. Emerging… Show more
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