The field of investigation about potential curative treatments for dystrophinopathies has evolved considerably in recent years, leading to multiple therapeutic strategies including gene therapy (exon skipping, micro-dystrophins, etc.)for restoration of dystrophin expression or increase the expression of utrophin protein, and treatments blocking the different pathophysiological mechanisms associated with the absence of dystrophin (oxidative stress, calcium homeostasis, NF-kB pathway, mitochondria dysfunction, etc). The gene discussed is UTRN; the disease is neuromuscular disease caused by qualitative or quantitative defects of dystrophin.