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Journal of Child Neurology
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Clinical Trials in Motor Neuron Diseases

Robert G. Miller, MD

California Pacific Medical Center, San Francisco, CA, rmiller{at}cooper.cpmc.org

Although there is no truly effective disease-specific therapy for any of the motor neuron diseases, rapid progress in our understanding of the pathophysiology of some of these disorders is being made. In addition to progress in neuroscience, clinical trials of agents that appear to slow the progress of at least one of these diseases, amyotrophic lateral sclerosis, are beginning to show promising results. The first clinical trials in spinal muscular atrophy are currently underway. A number of other developments have raised the quality of clinical trials, which should improve their productivity and efficiency in the future. (J Child Neurol 1999;14:173-179).

Journal of Child Neurology, Vol. 14, No. 3, 173-179 (1999)
DOI: 10.1177/088307389901400308
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L. P. Rowland and N. A. Shneider
Amyotrophic Lateral Sclerosis
N. Engl. J. Med., May 31, 2001; 344(22): 1688 - 1700.
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