Myotubular Myopathy

Myotubular Myopathy

A molecule for fighting muscular paralysis. Myotubular myopathy is a severe genetic disease that leads to muscle paralysis. Although no treatment currently exists, researchers have identified a molecule that not only greatly reduces the progression of the disease but also boosts life expectancy in animal models by a factor of seven. Since the molecule -- known as tamoxifen -- is already used for breast cancer, the researchers hope to soon set up a clinical trial. Source 1a.