Ravulizumab is approved for use in patients with paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), or anti-AChR antibody-positive gMG, and has been shown to provide complete terminal C5 complement inhibition throughout an 8-week dosing interval in these conditions [22–24]. The gene discussed is C5; the disease is paroxysmal nocturnal hemoglobinuria.