Abstract
All multicentre trials are challenging, whatever the disease under study. Motor neurone disease (MND; USA: amyotrophic lateral sclerosis, ALS) brings to the challenge a particular set of difficulties. Here is a condition that exists in different forms and presents extremely variably. It has its onset in a wide age range with, accordingly, a range of concomitant illnesses, progresses inexorably but somewhat unpredictably and again very variably, and offers no obvious manner of measurement. In pathophysiological terms, reversal of the condition is unattainable, arrest is the Holy Grail and slowed rate of progression the most realistic outcome to hope for. In other words, patients at the conclusion of a trial will be worse off than at entry.
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Steiner, T.J. (2001). Motor Neurone Disease: Experience with Large Multicentre Trials. In: Guiloff, R.J. (eds) Clinical Trials in Neurology. Springer, London. https://doi.org/10.1007/978-1-4471-3787-0_35
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DOI: https://doi.org/10.1007/978-1-4471-3787-0_35
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