State-Of-The-Art Advances in Duchenne Muscular Dystrophy

Abstract

Duchenne muscular dystrophy (DMD) is a severe and fatal muscle condition affecting young children. Without interventions, affected boys lose the ability to walk independently by the age of 10 and develop progressive cardiac and respiratory failure. The last 20 years have seen a change in the natural history of DMD following improvements in clinical care and proactive interventions to manage complications of the disease. An international collaboration of DMD experts has created care imperatives for best practice in DMD; these are now available in 30 different languages and are disseminated worldwide. An update of these care recommendations is currently under review. More recently, the field has seen encouraging scientific progress in regard to new therapeutic approaches of which a large number are currently being evaluated in clinical trials. With time, improvements in clinical care and access to new treatments and innovations are changing the natural course of DMD, from a relentless progressive illness with death in teenage years to a more chronic illness with a good quality of life and increased life expectancy. This is a particularly encouraging time for DMD, and experiences built in the muscular dystrophy field are likely to be of benefit to the development of new approaches and therapies in other rare diseases.