In a multicenter phase 2 trial involving 17 MM patients harboring mutationally activated or amplified KIT, Hody et al. [100] reported a 64% of ORR among those with KIT mutations (in exon 11, 13, and 17) treated with imatinib mesylate, whereas the drug was ineffective in patients only presenting KIT amplifications. Here, KIT is linked to Miyoshi myopathy.