Data from clinical trials demonstrated that the overall response rates (ORRs) from PD-1 blockade in MM from both the Asian (0%–13.3%)10 11 and Caucasian population (23.3%)12 were much lower than that of CM (33.7%–43.7%).13–15 Even the dual inhibition of PD-1 and CTLA-4 pathways yielded limited improvement in response rates (37.1%–43.0%) for patients with MM in the Caucasian population, with a median progression-free survival (PFS) of only 5.8–5.9 months.16 17. The gene discussed is CTLA4; the disease is Miyoshi myopathy.