Clinical Investigation of Duchenne Muscular Dystrophy: Interesting Results in a Trial of Prednisone

Michael H. Brooke, Gerald M. Fenichel, Robert C. Griggs, Jerry R. Mendell, Richard T. Moxley, J. Philip Miller, Kenneth K. Kaiser, Julaine M. Florence, Shree Pandya, Linda Signore, Wendy King, Jenny Robison, Richard A. Head, Michael A. Province, Warren Seyfried, Stephen Mandel

Research output: Contribution to journalArticlepeer-review

139 Scopus citations

Abstract

We investigated the effect of highdose prednisone therapy in 33 boys with Duchenne muscular dystrophy. The drug was given daily in doses of 1.5 mg/kg of body weight (to a maximum of 80 mg) for six months. Muscle strength, joint contractures, timed functional tests, functional ability, and pulmonary function were measured at the beginning and end of the treatment period. The trial was designed using natural history controls, and the power of the study was 0.80 to detect a slowing of 50% in the rate of progression. During the period of the trial, muscle strength, functional grades, timed functional tests, and pulmonary function improved. Contractures followed the expected natural history of the illness.

Original languageEnglish
Pages (from-to)812-817
Number of pages6
JournalArchives of neurology
Volume44
Issue number8
DOIs
StatePublished - Aug 1987

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