Amyotrophic lateral sclerosis (ALS) is a motor neurone disease (MND) characterized by death of motor neurons in the cerebral cortex, brain stem, and spinal cord. ALS is one of the most common adult motor neuron diseases that, at present, has no cure or effective treatment. Most of our current knowledge on the disease’s mechanisms has been obtained through studies of the familial forms induced by SOD1 mutations. The identification of mutations in VAP-B as cause for ALS has opened new opportunities for gaining insight into the underlying mechanisms of the disease and for developing new therapeutic avenues.
