ABSTRACT

The muscular dystrophies represent a group of slowly progressive inherited diseases that usually have a very specific pattern of muscle wasting and weakness. Because of better physical therapy, surgical, and ventilatory techniques, the lifespan of those patiets with these often progressive illnesses has grown in the last 50 years. With a few notable exceptions, however, current therapy for muscular dystrophies remains largely supportive and rarely targeted. Because of major advances in the diagnosis and treatment ofDuchenne dystrophy, this chapter will focus primarily on this dystrophinopathy. Briefer sections, as well as information contained in Table 1 address systemic treatment of some of the other muscular dystrophies that occur in childhood.