PEDIATRICS Vol. 36 No. 3 September 1965, pp. 402-405
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ANABOLIC STEROIDS IN THE MANAGEMENT OF MUSCULAR DYSTROPHY

Leon Charash M.D.1

1 Hospital for Special Surgery, affiliated with the New York Hospital–Cornell University Medical College, New York, New York

A group of 21 patients with muscular dystrophy, 17 of them boys with the pseudohypertrophic form, were treated with a regimen including the use of an anabolic steroid, digitoxin, and physical exercise. This program had previously been reported to be efficacious.

One year after the initiation of treatment all patients in the present study were significantly worse. None showed any change which could be interpreted as representing improvement. Five boys displayed rather rapid progression of the disease leading to loss of ambulation.

Physicians at eight other centers, with experience in trials of identical therapy in approximately 300 patients, report no evidence of any objective benefit from the use of this steroid therapy in muscular dystrophy.

Submitted on November 12, 1964
Accepted on March 10, 1965