CORDIS Project
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This project investigates the effects of Tamoxifen, a drug that mimics estrogen, on X-linked centronuclear myopathy, a severe muscle disorder. It aims to understand the drug's mechanisms and evaluate its efficacy in combination with other treatments in a mouse model of the disease.
X-linked centronuclear myopathy (XLCNM) is a severe congenital myopathy caused by the absence of lipid phosphatase myotubularin. XLCNM affects 1/50.000 male births, there is no treatment and many cases lead to premature death.
Thus, treatments are needed.Tamoxifen (TAM) is a selective estrogen receptor modulator that mimics estrogen signalling in skeletal muscle.
Pilot study and previous results from the hosting group, have shown that TAM improve muscle symptoms and survival rate in XLCNM mouse…
UNIVERSITE DE GENEVE
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