Drug Blocks Lethal Motor-Neuron Disease In Mice

... Spinal muscular atrophy (SMA) is an inherited motor-neuron disease that, in its most severe form, leads to death before 2 years of age and for which there is no treatment. It is caused by mutations in the gene SMN1 that prevent SMN1 producing the protein SMN. A second gene, SMN2, can also produce SMN, but it is much less efficient than SMN1 at doing so. [click link for full article] ...