Patients enrolled in the trial had anti-PD-1 or anti-PD-1/anti-CTLA-4-resistant cutaneous (n = 8), mucosal (n = 5), or uveal (n = 4) MM, and most patients were heavily pretreated with a median of 2 (1–7) prior cancer treatment lines. The gene discussed is CTLA4; the disease is Miyoshi myopathy.