Novel therapies for muscular dystrophy and other muscle wasting conditions

Abstract

The muscular dystrophies are generally characterised by progressive skeletal muscle wasting and weakness. Duchenne muscular dystrophy (DMD), an X-linked disorder, is the most severe of all the dystrophies and is caused by a variety of mutations and deletions in the dystrophin gene. In the absence of dystrophin expression, the skeletal muscles of boys with DMD undergo continuous cycles of degeneration and regeneration of muscle fibres that lead to a progressive wasting of the skeletal muscles. At age 10 years, DMD patients have only 25% of the muscle mass of healthy children and the functional burden on their weakened muscles is too great for normal ambulation well before this time. They beco..