CASC3 and Miyoshi myopathy: When combined with other therapeutic anticancer agents, BTZ could achieve even better clinical efficacy, thus leading to a full US FDA approval in 2005 as a second-line MM therapy [187,188], and as a first-line therapy for patients with newly diagnosed MM after only three years [189].Unfortunately, the therapeutic window of BTZ is relatively narrow and toxic side effects gradually started to appear, ranging from peripheral neuropathy, myelosuppression and cardiotoxicity.