Fenichel G, Pestronk A, Florence J, Robison V, Hemelt V
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.
PMID: 9153447, UI: 97297815
