Clinical investigation in duchenne dystrophy: 2. Determination of the “power” of therapeutic trials based on the natural history

Michael H. Brooke, Gerald M. Fenichel, Robert C. Griggs, Jerry R. Mendell, Richard Moxley, J. Philip Miller, Michael A. Province

Research output: Contribution to journalArticlepeer-review

298 Scopus citations

Abstract

A prospective study of 114 patients with DMD provided data for “power” calculations for future therapeutic trials. There was a decline in strength of 0.4 units per year (on a 0‐10 scale). Contractures of the iliotibial bands, hip flexors, and heel cords developed before 6 years. Contractures of other joints accompanied the increased use of wheelchairs. All children walked until 8 years with functional “improvement” between 3–6 years. Children of the same age varied widely in their strength, degree of contracture, and functional abilities. Fifteen percent of the patients appear to have a milder variety of the disease and are termed “outliers.” To test a drug which might slow the disease to 25% of its original rate of progression, two groups (placebo and treatment) of 40 patients each would have to be followed for one year.

Original languageEnglish
Pages (from-to)91-103
Number of pages13
JournalMuscle & Nerve
Volume6
Issue number2
DOIs
StatePublished - Feb 1983

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