Carrell-Krusen Symposium invited lecture. Clinical trials in motor neuron diseases.
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.
Miller
R G
RG
California Pacific Medical Center, San Francisco 94115, USA. rmiller@cooper.cpmc.org
D000085:Acetates / Q000627:therapeutic use; D000293:Adolescent; D000328:Adult; D000588:Amines; D000690:Amyotrophic Lateral Sclerosis / Q000188:drug therapy* / Q000235:genetics; D000818:Animals; D019208:Brain-Derived Neurotrophic Factor / Q000627:therapeutic use; D002648:Child; D002675:Child, Preschool; D002986:Clinical Trials as Topic / Q000592:standards*; D003509:Cyclohexanecarboxylic Acids; D018691:Excitatory Amino Acid Antagonists / Q000627:therapeutic use; D005260:Female; D000077206:Gabapentin; D018698:Glutamic Acid / Q000187:drug effects; D017408:Guidelines as Topic / Q000592:standards*; D006801:Humans; D007223:Infant; D007334:Insulin-Like Growth Factor I / Q000627:therapeutic use; D008297:Male; D051379:Mice; D008875:Middle Aged; D016472:Motor Neuron Disease / Q000188:drug therapy* / Q000235:genetics; D009134:Muscular Atrophy, Spinal / Q000175:diagnosis / Q000188:drug therapy* / Q000235:genetics; D009414:Nerve Growth Factors / Q000627:therapeutic use; D017063:Outcome Assessment, Health Care / Q000458:organization & administration; D016262:Postpoliomyelitis Syndrome / Q000188:drug therapy; D019782:Riluzole / Q000627:therapeutic use; D005680:gamma-Aminobutyric Acid
D000085:Acetates; D000588:Amines; D019208:Brain-Derived Neurotrophic Factor; D003509:Cyclohexanecarboxylic Acids; D018691:Excitatory Amino Acid Antagonists; D009414:Nerve Growth Factors; D018698:Glutamic Acid; D005680:gamma-Aminobutyric Acid; D007334:Insulin-Like Growth Factor I; D000077206:Gabapentin; D019782:Riluzole