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Clinical Investigation of Duchenne Muscular DystrophyInteresting Results in a Trial of Prednisone
Michael H. Brooke, MD;
Gerald M. Fenichel, MD;
Robert C. Griggs, MD;
Jerry R. Mendell, MD;
Richard T. Moxley III, MD;
J. Philip Miller;
Kenneth K. Kaiser;
Julaine M. Florence, MHS;
Shree Pandya, MS;
Linda Signore, BSN;
Wendy King;
Jenny Robison;
Richard A. Head, MD;
Michael A. Province, PhD;
Warren Seyfried, PhD;
Stephen Mandel
Arch Neurol. 1987;44(8):812-817.
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.
Author Affiliations
From the Washington University School of Medicine, St Louis (Drs Brooke, Head, Province, and Seyfried, Messrs Miller, Mandel and Kaiser, and Ms Florence); Vanderbilt University, Nashville, Tenn (Dr Fenichel and Ms Robison); University of Rochester (NY) (Drs Griggs, Moxley, and Ms Pandya); and Ohio State University, Columbus (Dr Mendell, and Mss Signore and King).
Footnotes
Accepted for publication April 15, 1987.
Reprint requests to Department of Neurology, Washington University Medical School, 660 S Euclid, Box 8111, St Louis, MO 63110 (Dr Brooke).
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