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

Gerald M. Fenichel, A. Pestronk, J. Florence, V. Robison, V. Hemelt

Research output: Contribution to journalArticlepeer-review

49 Scopus citations

Abstract

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.

Original languageEnglish
Pages (from-to)1225-1226
Number of pages2
JournalNeurology
Volume48
Issue number5
DOIs
StatePublished - May 1997

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