Duchenne muscular dystrophy is the most common neuromuscular disorder of childhood with an incidence of 1 in 3,500 live male births, and although significant advances have been made in the past 25 years in our understanding of the molecular genetics of the disease, no cure has been found. Duchenne muscular dystrophy presents a multitude of challenges for drug discovery and for the development of effective treatments. A number of promising therapeutic approaches have been developed which are aimed at replacing the missing dystrophin (or an alternative compensatory protein), or identifying drugs that work on molecular pathways that may reduce the severity of the disease. Unfortunately, we are not advancing fast enough and our children, our future is suffering greatly.



