this mechanism of action enables eculizumab to be considered in the treatment of individuals who have aHUS associated with defects in both soluble and membrane-bound complement regulators.

Such cases are known as atypical HUS (aHUS) cases. 1 The cardinal feature of aHUS is dysregulation of the alternative complement pathway, and gene mutations are found in more than 50% of cases.

Alternative pathway dysregulation is a central event in development of certain diseases caused by mutations or polymorphisms in complement regulator factor H (FH). While mutations in the C-terminal ...