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Tamoxifen Shows Pre-Clinical Potential in Muscular Dystrophy

Drug improves muscle strength and function in mouse model

THURSDAY, Jan. 17 (HealthDay News) -- The breast cancer drug tamoxifen improves muscle strength and function in a mouse model of Duchenne muscular dystrophy (DMD), according to a study published online Jan. 15 in The American Journal of Pathology.

Noting that tamoxifen affects processes that contribute to the pathogenesis of DMD, Olivier M. Dorchies, Ph.D., from the University of Geneva, and colleagues treated mdx5Cv mice, a model of DMD, with 10 mg/kg/day oral tamoxifen starting at 3 weeks of age for 15 months.

The researchers found that tamoxifen treatment stabilized myofiber membranes, increased whole body strength to normal values, and improved the structure of leg muscles. Tamoxifen also reduced cardiac fibrosis by ~50 percent and increased the amount of contractile tissue in the diaphragm available for respiration by 72 percent. In addition, muscles had a slower rate of contraction and greater resistance to fatigue.

"In conclusion, our preclinical evaluation of tamoxifen in a mouse model of DMD showed promising improvements of skeletal and cardiac muscles," Dorchies and colleagues write. "However, more investigations are required to establish the actions of tamoxifen on further aspects of the dystrophic disease, such as the prevention of the initial muscle necrosis and the modulation of the inflammatory responses."

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