The most promising investigational new agents with significant single-agent activity in MM include isatuximab, an anti-CD-38 monoclonal antibody; marizomib, a new proteasome inhibitor; oprozomib, an oral proteasome inhibitor related to carfilzomib; filanesib (ARRY-520), a kinesin spindle protein (KSP) inhibitor; dinaciclib, a cyclin-dependent kinase (CDK) inhibitor; venetoclax (ABT-199), a selective BCL-2 inhibitor; and LGH-447, pan PIM kinase inhibitor (Table 1). The gene discussed is CD38; the disease is Miyoshi myopathy.