Oxandrolone is a synthetic anabolic steroid that mimics the naturally occurring male sex hormone testosterone.

How oxandrolone works

The mechanism of action of oxandrolone is similar to that of other androgen hormones such as testosterone. It binds to androgen receptors in the cytoplasm of muscle cells. The androgen-receptor-oxandrolone complex then translocates to the nucleus and binds to genes that contain androgen-receptor binding sites that promote the transcription of several genes whose protein products are necessary for muscle growth, metabolism, repair, and maintenance.

In this way, oxandrolone may potentially prevent muscle degeneration in muscular dystrophy patients, helping them to maintain their strength and prolong their ability to walk and carry out daily activities.

Oxandrolone in clinical trials

A pilot study enrolled 10 boys with Duchenne muscular dystrophy (DMD) to assess the effect of three months of oxandrolone treatment. The results showed a mild improvement in the muscle score of the boys, suggesting that oxandrolone may be beneficial in the treatment of DMD.

A six-month, randomized, double-blind, placebo-controlled study in boys with DMD showed no significant change in the average manual muscle strength score, compared with placebo controls, but the boys did show significant improvements in quantitative muscle tests. This suggested that oxandrolone treatment may help in slowing muscle weakness in these patients.

Another study reported that when four boys diagnosed with DMD were treated with 0.1 mg per kg of body weight per day of oxandrolone for three months, there was a significant increase in the rate of muscle protein synthesis. Gene expression analysis of their muscle specimens obtained before and after treatment revealed a decrease in muscle regeneration upon oxandrolone treatment, likely as a result of a decrease in muscle degeneration.

A randomized, double-blind, clinical trial (NCT00027391) tested whether treatment with albuterol or oxandrolone, alone or in combination, for 52 weeks can increase strength and muscle mass in 160 patients with facioscapulohumeral dystrophy. This study started in September 2001 and was completed in August 2004. However, the results have not been published.

Other information

Oxandrolone is currently not recommended for long-term treatment in DMD patients.

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