This agent demonstrated high antitumoral activity in micromolar concentrations in drug-sensitive and -resistant MM cell lines and MM patients, due to the well-known activation of proapoptotic Bax and depolarization with an increased permeability of the mitochondrial membrane, followed by cytochrome c and SMAC/direct inhibitor of apoptosis-binding protein with low isoelectric point (DIABLO) translocation into the cytosol, finally resulting in the activation of caspase-8, -9, and -3. The gene discussed is BAX; the disease is Miyoshi myopathy.