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Neurology. 1997 May;48(5):1225-6.

A beneficial effect of oxandrolone in the treatment of Duchenne muscular dystrophy: a pilot study.

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Department of Neurology, Vanderbilt University School of Medicine, Nashville, TN 37212, USA.


We treated 10 boys with Duchenne muscular dystrophy with oxandrolone, an anabolic steroid, for 3 months. The mean of the changes in the average muscle score was an improvement of 0.315 +/- 0.097. The expected mean change in muscle score after 3 months from natural history data is a loss of 0.1. The difference of 0.415 between the actual and expected values is significant at p < 0.01. This preliminary evidence suggests that oxandrolone treatment may provide a degree of functional benefit for children with Duchenne muscular dystrophy that is similar to that after prednisone administration.

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