A beneficial effect of oxandrolone in the treatment of Duchenne muscular dystrophy: a pilot study.
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.
Fenichel
G
G
Department of Neurology, Vanderbilt University School of Medicine, Nashville, TN 37212, USA.
Pestronk
A
A
Florence
J
J
Robison
V
V
Hemelt
V
V
D045930:Anabolic Agents / Q000627:therapeutic use*; D002648:Child; D006801:Humans; D008297:Male; D009136:Muscular Dystrophies / Q000188:drug therapy* / Q000503:physiopathology; D010074:Oxandrolone / Q000009:adverse effects / Q000627:therapeutic use*; D010865:Pilot Projects; D016896:Treatment Outcome
D045930:Anabolic Agents; D010074:Oxandrolone