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  1. Paroxysmal nocturnal hemoglobinuria

    ... congenital anomalies-hypotonia-seizures syndrome 2. Eculizumab , first-in-class monoclonal antibody that inhibits ... should be reserved for patients with suboptimal response to eculizumab. Bone Marrow ...

    Research Article last updated 10/02/2014 - 8:35am.

  2. Complement Blockade with a C1 Esterase Inhibitor in Paroxysmal Nocturnal Hemoglobinuria

    ... context of underlying complement activation. Currently eculizumab effectively blocks the intravascular hemolysis PNH. There ... products on CD55 deficient erythrocytes from PNH patient on eculizumab therapy. This could suggest a role for inhibition of earlier phases ...

    Research Article last updated 08/04/2014 - 8:13am.

  3. Anti-Complement Treatment in Paroxysmal Nocturnal Hemoglobinuria: Where we Stand and Where we are Going

    ... resulting in erythrocyte lysis. The availability of eculizumab as the first complement inhibitor for clinical use renewed the ... independence in at least half of PNH patients receiving eculizumab, with adequate control of all hemolysis-associated symptoms even in ...

    Research Article last updated 05/22/2014 - 8:24am.

  4. Kimberly Woudenberg – Knowing What it Takes

    ... I had to face how this disease affected me. I started eculizumab (Soliris®) and my blood counts stabilized, but remained ...

    Patient Chronicle last updated 05/18/2015 - 2:57pm.

  5. A Story of Hope: Emma Pritchett

    ... regularly sees her local hematologist.  “Soliris ® ( eculizumab ) has been a life-changer,” she said.  “Things are so much ...

    Patient Chronicle last updated 05/18/2015 - 2:56pm.

  6. Long-term outcome of fludarabine-based reduced intensity allogeneic hematopoietic cell transplantation for debilitating paroxysmal nocturnal hemoglobinuria

    ... option for PNH patients who are not candidates for eculizumab treatment. Bone ...

    Research Article last updated 06/02/2014 - 11:39am.

  7. Baseline characteristics and disease burden in patients in the international paroxysmal nocturnal hemoglobinuria registry

    ... to evaluating the long-term safety and effectiveness of eculizumab in a global population, the registry aims to improve diagnosis, ... anticoagulation (31%), immunosuppression (19%), and eculizumab (25%). Frequently reported symptoms included fatigue (80%), dyspnea ...

    Research Article last updated 03/06/2014 - 11:30am.

  8. Peptide inhibitors of C3 activation as a novel strategy of complement inhibition for the treatment of paroxysmal nocturnal hemoglobinuria

    ... and CD59 on affected erythrocytes. The anti-C5 antibody eculizumab has proven clinically effective, but uncontrolled C3 activation ...

    Research Article last updated 03/06/2014 - 11:17am.

  9. Paroxysmal nocturnal hemoglobinuria and the age of therapeutic complement inhibition

    ... mortality, or treatment with the complement inhibitor eculizumab . Eculizumab is a first-in-class anti-complement drug that in PNH has been shown ...

    Research Article last updated 11/05/2013 - 12:37pm.

  10. Selective splenic artery embolization for the treatment of thrombocytopenia and hypersplenism in paroxysmal nocturnal hemoglobinuria

    ... One patient, who had had only a partial response to eculizumab , responded to SSAE not only with an improved platelet count, ... red cells in patients who have had a limited response to eculizumab, resulting in an improved quality of life for selected patients. ...

    Research Article last updated 04/17/2014 - 1:34pm.