Definition of "dystrophin" [dystrophin]

• A cytoplasmic structural protein that is deficient in some forms of muscular dystrophy (noun)

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• "•It is caused by an absence of a protein called dystrophin, which helps to keep the cells found in muscles intact."
• "In muscular dystrophy, the loss of a protein called dystrophin causes the muscle to literally tear itself apart, a process that cannot be repaired without cell-based intervention."
• "This study is a Phase 2b, multicenter, randomized, double-blind, placebo-controlled, dose-ranging, efficacy and safety study, designed to document the clinical benefit of PTC124 when administered as therapy of patients with DMD/BMD due to a nonsense mutation (premature stop codon) in the dystrophin gene."