PATHOPHYSIOLOGY OF MUSCULAR-DYSTROPHY

The inevitable and remorseless clinical decline in a patient with Duchenne muscular dystrophy (DMD) evokes a special sense of impotent desperation, which was, until recently, exacerbated by lack of obvious progress in its treatment. However, identification of the culpable gene has provided insight into the pathogenesis of DMD and glimmerings of hope for its treatment.