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Open Access Repurposing the Selective Oestrogen Receptor Modulator Tamoxifen for the Treatment of Duchenne Muscular Dystrophy

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Drug discovery is a long, expensive and risky process. Evaluating drugs that have already been proved safe for use in humans and testing them for a new indication greatly reduces the time and monetary costs involved in finding treatments for life-threatening conditions. Here tamoxifen, a drug that is used for the treatment of breast cancer, is investigated in a mouse model of Duchenne muscular dystrophy. Tamoxifen was efficacious in countering the symptoms of the disease without affecting the underlying genetic cause. Based on these results, tamoxifen has been tested in other forms of muscle disease with success. Drug repurposing may not only be a cost-effective manner for treating a variety of diseases, it may also help us uncover common mechanisms between conditions that were previously thought to be unrelated.

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Keywords: DUCHENNE MUSCULAR DYSTROPHY; OESTROGENS; REPURPOSING; SERM; TAMOXIFEN

Document Type: Research Article

Affiliations: 1: School of Pharmaceutical Sciences, University Medical Center University of Geneva, CMU 5-6 Rue Michel-Servet 1, Geneva, Switzerland 2: School of Pharmaceutical Sciences, University Medical Center University of Geneva, CMU 5-6 Rue Michel-Servet 1, Geneva, Switzerland. [email protected]

Publication date: April 1, 2018

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  • International Journal for Chemistry and Official Membership Journal of the Swiss Chemical Society (SCS) and its Divisions

    CHIMIA, a scientific journal for chemistry in the broadest sense, is published 10 times a year and covers the interests of a wide and diverse readership. Contributions from all fields of chemistry and related areas are considered for publication in the form of Review Articles and Notes. A characteristic feature of CHIMIA are the thematic issues, each devoted to an area of great current significance.

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