Periodic Reporting for period 1 - TAMYOCAL (Tamoxifen mediated protection on X-linked centronuclear myopathy: a mechanistic and pre-clinical study)

X-linked myotubular myopathy (XLMTM) is a severe congenital myopathy caused by the absence of lipid phosphatase myotubularin. XLMTM 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...