However, the study was designed only to test safety, not the effectiveness of the treatment aimed at the 13, 500 Americans who have severe hemophilia A, a form of the genetic disease that causes excessive bleeding.
32.
Factor VIII turned out to be deficient in the clinically recognised but etiologically elusive hemophilia A; it was identified in the 1950s and is alternatively called " antihemophilic globulin " due to its capability to correct hemophilia A.
33.
One of the keys to understanding the rationale behind the Bayer-Avigen collaboration is visible from Interstate 80 in Berkeley, Calif ., where Bayer employs 1, 400 people at a plant that makes the clotting proteins for hemophilia A.
34.
Factor VIII turned out to be deficient in the clinically recognised but etiologically elusive hemophilia A; it was identified in the 1950s and is alternatively called " antihemophilic globulin " due to its capability to correct hemophilia A.
35.
"These are the first positive results from a trial of gene therapy for hemophilia A, " which causes abnormal bleeding in about 13, 500 Americans, said Dr . David Roth of Beth Israel Deaconess Medical Center, the principal investigator.
36.
The Chiron Corporation, a biotechnology company based in Emeryville, Calif ., has treated eight hemophilia A patients with a gene therapy using a retrovirus as a vector, and plans to release its first data by the middle of next year.
37.
The recently disclosed files also contained a June 7 memo on blood products for Hemophilia A, which stated that if the Japanese Red Cross provided 44 percent of plasma to medical institutions, an ample supply of safer domestic blood products would be made available.
38.
Hemophilia B, less common than hemophilia A, is a gender-linked bleeding disorder caused by the absence or deficiency of the blood clotting protein factor IX . According to the World Hemophilia Foundation, one in 10, 000 males born worldwide has hemophilia, which afflicts about 400, 000 people worldwide.
39.
The usefulness of desmopressin in hemophilia A depends predominantly on whether the deficiency involved is qualitative ( i . e ., there is a functional mutation of the protein itself ) or quantitative ( i . e ., there is decreased production of a functionally normal protein ).
40.
Factor XII does play an important role in clot formation during " in vitro " measurements of the partial thromboplastin time, which causes these measurements to be markedly prolonged in patients with factor XII deficiency, usually well beyond even what is seen in hemophilia A, hemophilia B, or factor XI deficiency.
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