Eculizumab Shows Efficacy and Tolerability in Pediatric Patients with PNH

An open-label study of seven pediatric patients with paroxysmal nocturnal hemoglobinuria (PNH) found short-term infusions of eculizumab to be well tolerated, with reduced intravascular hemolysis. These results, reported during the 53rd American Society of Hematology Annual Meeting and Exposition, were consistent with those found in adults with this disease and suggest the potential for this treatment in children and adolescents ...

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