Systemic Treatment of Body‐Wide Duchenne Muscular Dystrophy Symptoms
Patryk Konieczny
Abstract: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 adverse effects and even death. Likewise, it is now clear that the absence of dystrophin affects the fu… Show more
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