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NEUROLOGY 1997;48:1225-1226
© 1997 American Academy of Neurology

A beneficial effect of oxandrolone in the treatment of Duchenne muscular dystrophy

A pilot study

G. Fenichel, MD, A. Pestronk, MD, J. Florence, MS, V. Robison, BS and V. Hemelt, MD

From the Departments of Neurology, Vanderbilt University School of Medicine (Dr. Fenichel and V. Robison), Nashville, TN, and Washington University School of Medicine (Drs. Pestronk and Hemelt, and J. Florence), St. Louis, MO.

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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