Duchenne muscular dystrophy is a devastating inherited neuromuscular disorder that affects one in 3.500 live male births. It is caused by mutations of the DMD gene, leading to progressive muscle weakness, loss of independent ambulation by early teens, and premature death due to cardiorespiratory complications. Pharmacological intervention has begun to change the natural history of DMD, and further advances and more effective treatment of the underlying pathology of DMD should continue to offer an improved course, potentially including small-molecule and gene therapies. The most devastating and obvious effect of DMD is on the skeletal musculature with resulting loss of strength and function. Glucocorticoids are the only medication currently available that slows the decline in muscle strength and function in DMD which in turn reduces the risk of scoliosis and stabilises pulmonary function. Cardiac function might also improve, with limited data to date indicating a slower decline in echocardiographic measures of cardiac dysfunction, although these measures are not necessarily predictive of the delay in cardiac symptoms, signs, or cardiac-related mortality. The main therapeutic strategies include: 1) gene replacement or other genetic therapies linked to specific mutations to restore dystrophin production; 2) membrane stabilization and/or upregulation of compensatory proteins; 3) reduction of the inflammatory cascade and/or enhancement of muscle regeneration. Specific examples of emerging therapeutic strategies for DMD are exon skipping and nonsense mutation suppression therapies. Exon skipping uses synthetic antisense oligonucleotide sequences to correct specific dystrophin gene mutations. It does so by inducing specific exons skipping during premessenger RNA splicing of the dystrophin gene, resulting in restoration of the reading frame and partial production of an internally truncated protein. The first drug that has passed the phases of clinical trials is EXONDYS 51 (Eteplirsen), an antisense oligonucleotide that is designed to bind to exon 51 of dystrophin pre-mRNA, resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 51 skipping.The first muscular dystrophy patient in the world to receive Exondys 51 is seven-year-old Wyatt Hubbard from Ohio,USA.  In conclusion, the precise correction of dystrophin mutations or the splicing out of the exon containing such a mutation holds promise, although these methods require optimization for almost every different mutation in the gene.