ABSTRACT
There is no cure currently available for Duchenne muscular dystrophy (DMD), a devastating and relentlessly progressive X-linked recessive muscle disease with an incidence of 1 in 3500 male births. In this condition, dystrophin is already absent in fetal muscle biopsies and creatine kinase (CK) levels are markedly elevated at birth, signifying active muscle degeneration and regeneration (1-3). Remarkably, affected boys are normal at birth and over the first one or two years of life, with overt clinical weakness often not evident for three or four years. Typically, weakness manifests before five years of age as difficulty in walking, running, or rising from the floor, followed by progression to the use of a wheelchair in the early teens and death due to cardiorespiratory failure in the second or third decade (see Chapter 1).
