[Tamoxifen, a high-potential molecule to treat all centronuclear myopathies].

Med Sci (Paris)

IGBMC - CNRS UMR 7104 - Inserm U 1258, Illkirch, France.

Published: November 2024

Centronuclear myopathies are rare congenital disorders characterized by muscle weakness and mislocalization of organelles. The main genes associated to these muscle diseases are MTM1, DNM2, BIN1 and RYR1. To date, no therapy is available. Nevertheless, tamoxifen, a pharmacological compound already used in clinics for breast cancer, showed beneficial effects on the muscle phenotypes in mouse models for centronuclear myopathies. Here, the effects of tamoxifen on muscle phenotypes will be compared in the various forms of this muscle disease.

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Source
http://dx.doi.org/10.1051/medsci/2024130DOI Listing

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