Abstract
aHUS is a rare disease characterized by a dysregulated and overactive complement system. Prior to the introduction of eculizumab, a complement C5-inhibitor, outcomes for patients with aHUS were generally poor. Eculizumab has substantially improved patient prognosis, but requires biweekly infusions that can impose a large treatment burden for patients and caregivers. Ravulizumab was designed via targeted modification of eculizumab to reduce dosing frequency to every 4 to 8 weeks, with an immediate, complete and sustained inhibition of complement C5.
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