PNH Patients Treated with Soliris(TM) Experienced Dramatic Reduction in Blood Clots During Clini... (The article is availableonline at PNH...)

PNH Patients Treated with Soliris(TM) Experienced Dramatic Reduction in Blood Clots During Clinical Trials

Date:8/20/2007

The article is available online at http://bloodjournal.hematologylibrary.org/cgi/content/abstract/blood-2007-0 6- 095646v1. The analysis will also be published in a future print edition of Blood.

About PNH

PNH is an acquired genetic blood disorder defined by hemolysis, in which patients' red blood cells are destroyed by complement, a component of the body's immune system. PNH is a rare disease that affects an estimated 8,000 to 10,000 people in North America and Europe.(13) PNH often strikes people in the prime of their lives, with an average age of onset in the early 30's.(3) Approximately ten percent of all patients first develop symptoms at 21 years of age or younger.(2) PNH develops without warning and can occur in men and women of all races, backgrounds and ages. PNH often goes unrecognized, with delays in diagnosis often ranging from one to more than 10 years.(4) The estimated median survival for PNH patients is between 10 and 15 years from the time of diagnosis.(13,4)

PNH has been identified more commonly among patients with disorders of the bone marrow, including aplastic anemia (AA) and myelodysplastic syndrome (MDS).(14,15,16,17) In patients with thrombosis of unknown origin, PNH may be an underlying cause.(2,18)

Prior to approval of Soliris, there were no therapies specifically available for the treatment of PNH. PNH treatment was limited to symptom management through periodic blood transfusions, non-specific immunosuppressive therapy and, infrequently, bone marrow transplantations -- a procedure that carries considerable mortality risk.(2,18)

About Soliris

Soliris was approved in March 2007 by the U.S. Food and Drug Administration (FDA) as the first treatment for PNH, a rare, debilitating and life-threatening blood disorder defined by hemolysis, or the destruction of red blood cells. In June 2007, the European Commi
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SOURCE Alexion Pharmaceuticals, Inc.

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PNH Patients Treated with Soliris(TM) Experienced Dramatic Reduction in Blood Clots During Clini...(The article is availableonline at PNH...)