Clinical investigation in duchenne dystrophy: 2. Determination of the “power” of therapeutic trials based on the natural history
作者： Michael H. Brooke , Gerald M. Fenichel , Robert C. Griggs , Jerry R. Mendell , Richard Moxley
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摘要: A prospective study of 114 patients with DMD provided data for "power" calculations future therapeutic trials. There was a decline in strength 0.4 units per year (on 0-10 scale). Contractures the iliotibial bands, hip flexors, and heel cords developed before 6 years. other joints accompanied increased use wheelchairs. All children walked until 8 years functional "improvement" between 3-6 Children same age varied widely their strength, degree contracture, abilities. Fifteen percent appear to have milder variety disease are termed "outliers." To test drug which might slow 25% its original rate progression, two groups (placebo treatment) 40 each would be followed one year.
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