X-Linked centronuclear myopathy (XLCNM) is a rare and severe congenital myopathy characterised by generalised muscle weakness and abnormal nuclei positioning. Most affected boys die in their first year of life and survivors fail to achieve independent ambulation. It is caused by mutations in the Mtm1 gene encoding myotubularin, a ubiquitously expressed phosphoinositide phosphatase. No cure exists and very few pharmacological avenues are being explored. Here, we treated Mtm1-null mice with tamoxifen (TAM), a drug that modulates estrogen actions and that we have shown earlier to be efficacious in dystrophic (mdx5Cv) mice, a model of Duchenne muscular dystrophy (DMD).

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