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We are a biopharmaceutical company focused on serving patients with devastating and ultra-rare disorders through the innovation, development and commercialization of life-transforming therapeutic products. In our complement franchise, Soliris® is the first and only therapeutic approved for patients with either paroxysmal nocturnal hemoglobinuria (PNH), a life-threatening and ultra-rare genetic blood disorder, or atypical hemolytic uremic syndrome (aHUS), a life-threatening and ultra-rare genetic disease.
PNH and aHUS result from chronic uncontrolled activation of the complement component of the immune system. In our metabolic franchise, we commercialize Strensiq® for the treatment of patients with Hypophosphatasia (HPP) and Kanuma® for the treatment of patients with Lysosomal Acid Lipase Deficiency.