Transition and outcomes for young men with Duchenne muscular dystrophy in New South Wales

Abstract

Duchenne muscular dystrophy (DMD) is a progressive disorder that affects 1 in 3500 males and that typically results in death between 20 and 35years of age from respiratory or cardiac failure. International standards of care have been devised to guide the management of DMD patients. Due to its high incidence in all populations of the world and relatively homogeneous natural history, health outcomes in DMD provide a useful yardstick by which to assess neuromuscular services between regions. There is currently little data available on how males with DMD in NSW, Australia, fare in terms of health outcomes. Such data would provide health service providers important feedback about the effectivenes..