ABSTRACT

Neuromuscular disorders have been at the vanguard of the transition of the clinical neurosciences from descriptive to mechanistic. The hereditary muscular dystrophies provided fertile ground for modern molecular genetics to define aetiology, and for cell biology to determine pathogenesis. Similar advances have been made in the channelopathies and myotonias. Thus neuromuscular disorders have become one of the most exciting and rapidly advancing areas of clinical neurology. While molecular genetic analysis has allowed genetic counselling, specific treatments have been slower to develop. Nevertheless, it is in the area of gene therapy that muscle disease once again leads the way forward. There are grounds for optimism that the next 20 years will see

the development of successful therapies for many of these degenerative disorders.