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Amyotrophic lateral sclerosis (ALS) is a rapidly progressive neurodegenerative disorder resulting in loss of motor neurons within the motor cortex and bulbospinal regions. The worldwide prevalence is around 4–6 per 100 000, and the disease generally carries a poor prognosis with respiratory failure and death usually within 3 years, although the disease course can be highly variable.1 At present, there is no proven effective treatment for ALS aside from riluzole, a modulator of glutaminergic and Na+ pathways, which confers at most a modest improvement in survival by about 3 months through its disease modifying effects.2 ,3 The heterogeneity in clinical presentation, natural history and prognosis combined with the lack of definitive diagnostic tools, and hence considerable diagnostic delays contribute to the challenges in establishing effective drug trials in ALS.
Currently, the pathogenesis of ALS remains poorly understood, but is likely to be a complex interplay between polygenetic, environmental and lifestyle factors …
Competing interests None declared.
Provenance and peer review Commissioned; internally peer reviewed.