Systemic Treatment of Body-Wide Duchenne Muscular Dystrophy Symptoms.

Abstract excerpt

Duchenne muscular dystrophy (DMD) is a fatal X-linked disease that leads to premature death due to the loss of dystrophin. Current strategies predominantly focus on the therapeutic treatment of affected skeletal muscle tissue. However, certain results point to the fact that with successful treatment of skeletal muscle, DMD-exposed latent phenotypes in tissues, such as cardiac and smooth muscle, might lead to...

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