The almost complete loss of dystrophin isoform Dp427-M and concomitant reduction in dystrophin-associated proteins triggers the complex pathophysiology of Duchenne muscular dystrophy [7,25], an X-linked and multifaceted disorder of early childhood that primarily affects striated muscles [26], causing respiratory dysfunction, late-onset cardiomyopathy and scoliosis. The gene discussed is DMD; the disease is Duchenne muscular dystrophy.