10. Dantrolene as a Therapeutic Option for Wolfram Syndrome (134-OR)

Wolfram syndrome is a genetic disorder characterized by diabetes and neurodegeneration and considered as an endoplasmic reticulum (ER) disease. Despite the underlying importance of ER dysfunction in Wolfram syndrome and the identification of two causative genes, WFS1 and WFS2, a molecular mechanism linking the ER to death of β cells and neurons was not clear. We have recently discovered that calpain 2 is involved in death of β cells and neurons in Wolfram syndrome.