Ongoing trials in motor neurone disease

Abstract

Motor neurone disease (MND) is a group of progressive neurodegenerative disorders that cause disability from weakness and lead to death from respiratory failure. The pathophysiology of the several forms of MND is unknown, but recent advances have led to clinical trials of therapeutic agents based on an improved understanding of the pathologic processes. The design of clinical trials in MND is challenging, because an effective drug cannot restore strength, but rather slow the rate of progression. Measurement of progression poses difficulties, and an optimum end-point measure has not been determined. This article will include the clinical features of MND, present the leading hypotheses about causes as they relate to drug therapy, discuss factors to consider in selecting informative end-point measures, and will review past and current drug trials.

• amyotrophic lateral sclerosis
• drug trials
• end-point measures
• motor neurone disease
• spinal muscular atrophy