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Therapy brings blood disorder hope


Soliris (eculizumab) can help treat patients with a rare blood disorder, according to new research.

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, debilitating and life-threatening condition, defined by the destruction of red blood cells, or hemolysis.

In an ongoing study by Washington University, Soliris, a complement inhibitor, reduced hemolysis, fatigue, blood clots and transfusion requirements in PNH sufferers.

The study also shows that patients who might have been expected to have less severe forms of the disease suffer from significant problems.

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Dr Monica Bessler, lead author of the study, said: “PNH patients once thought to have less severe disease based on their clinical characteristics actually face significant disease burden from anaemia, fatigue, impaired quality of life, blood transfusion requirements and blood clot risk.

“The results show that, in this study group, regardless of disease severity, long-term Soliris treatment provides important clinical improvements in their disease signs, symptoms and complications.”

Soliris, developed by Alexion Pharmaceuticals, is the first therapy approved for the treatment of patients with PNH.

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Washington University

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