The inhibition of terminal complement component 5 (C5) is approved by the Food and Drug Administration (FDA)- and/or the European Medicine Agency (EMA) for the treatment of paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), and more recently to treat refractory generalized myasthenia gravis (rgMG). The gene discussed is C5; the disease is atypical hemolytic-uremic syndrome.