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Across the clinical neurosciences, the accurate measurement of disease burden remains a priority, not solely to facilitate clinical trial design, but also to accelerate therapeutic pipelines. Nowhere is the need greater than in amyotrophic lateral sclerosis (ALS), for which clinical trials have long been limited by problems in choosing reliable end points.1 Early trials focused on mortality but these required a definition of death—how should patients managed with ventilatory support or supported by gastroenterostomy be classified? Arbitrary ventilatory end points, such as forced expiratory volume, require skilled observers and practice to ensure uniformity and can only measure function in the neuromuscular system concerned with ventilation. Quality of life measures are perhaps even more nebulous, reflecting emotional state, which may be further biased by frontotemporal dementia. The most commonly utilised measure, the ALS Functional Rating Scale, with an added ventilatory component, the ALSFRS-v, is an overall, non-linear scale made up of questions related to bulbar, upper limb and lower limb function.2 It is smoothed by its presentation as a simple additive score, thus including bodily functions much affected and little …
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