Advances in neuromuscular disease 2003: the year in review.

During 2003, numerous research advances in both clinical neuromuscular disease and in the basic pathophysiology of these disorders were published and/or presented. In this review, we present a few categorical highlights of the year, discussing a new potential treatment of McArdle's disease, proposed new diagnostic criteria for the inflammatory myopathies and their clinical implications, the emerging anti-MuSK antibody syndrome in patients with myasthenia gravis, potential new therapies for the most common hereditary neuropathy (Charcot-Marie-Tooth type 1A), the successful pharmacologic manipulation and its therapeutic implications of the genetic mechanisms underlying spinal muscular atrophy, and several emerging therapeutic strategies in amyotrophic lateral sclerosis. As these reports indicate, clinical and basic research in neuromuscular disease continues to yield important and clinically relevant insights, which are now being rapidly translated into new clinical trials showing therapeutic promise for diseases previously thought untreatable.