For example, the HAVEN 7 clinical trial primary analysis (NCT04431726; published after the cut-off date) showed that over 52 weeks, emicizumab was efficacious and well tolerated in 55 infants with severe hemophilia A without FVIII inhibitors (median [range] treatment duration, 100.3 [52-118] weeks); 54.5% of participants had zero treated bleeds and no deaths, intracranial hemorrhages, thrombotic events, or thrombotic microangiopathy were reported [22]. This evidence concerns the gene F8 and Genetic thrombotic microangiopathy.