[Articles] Safety and efficacy of tamoxifen in boys with Duchenne muscular dystrophy (TAMDMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial

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A study found that tamoxifen was safe but did not show any significant difference in effectiveness compared to a placebo for treating Duchenne muscular dystrophy in boys. Therefore, the use of tamoxifen cannot be recommended as a treatment option due to insufficient clinical evidence.

Tamoxifen was safe and well tolerated, but no difference between groups was reported for the primary efficacy endpoint. Slower disease progression, defined by loss of motor function over time, was indicated in the tamoxifen group compared with the placebo group, but differences in outcome measures were neither clinically nor statistically significant. Currently, we cannot recommend the use of tamoxifen in daily clinical practice as a treatment option for boys with Duchenne muscular dystrophy due to insufficient clinical evidence.

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