Abstract
For over a century, the clinical and pathological essentials of motor neuron degeneration have been described [1]. Yet to this date, no cogent, easily applied quantiative description of the course of motor neuron degeneration has been forthcoming [2]. A complete understanding of the clinical features of motor neuron disease, both in the individual and within a group of affected individuals, is necessary for the optimal conduct of therapeutic trials. Precise endpoints are required to permit statistical planning of clinical trials, which will include determination of sample size, trial duration, and mode of analysis. In this chapter we will review the strategies to be applied when determining the best measures of change in individual patients or groups of patients with motor neuron disease over different time periods. We will also analyze the different statistical needs of individual case studies and parallel placebo-controlled case studies.
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Brooks, B.R., Depaul, R., De Tan, Y., Sanjak, M., Sufit, R.L., Robbins, J. (1990). Motor Neuron Disease. In: Porter, R.J., Schoenberg, B.S. (eds) Controlled Clinical Trials in Neurological Disease. Foundations of Neurology, vol 1. Springer, Boston, MA. https://doi.org/10.1007/978-1-4613-1495-0_18
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DOI: https://doi.org/10.1007/978-1-4613-1495-0_18
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