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Bleeding Disorder Blog
Related terms: Coagulopathy, Consumption coagulopathy, Disseminated Intravascular Coagulation (DIC), DIC
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FDA Approves Corifact to Prevent Bleeding in People With Rare Genetic Defect
Posted 21 Feb 2011 by Drugs.com
SILVER SPRING, Md., Feb. 17, 2011 /PRNewswire-USNewswire/ – The U.S. Food and Drug Administration today approved Corifact, the first product intended to prevent bleeding in people with the rare genetic defect congenital Factor XIII deficiency. Patients with congenital Factor XIII deficiency don't make enough Factor XIII, a substance that circulates in the blood and is important for normal clotting. Without treatment, people with the condition are at risk for life-threatening bleeding. Congenital Factor XIII deficiency is rare and affects 1 out of every 3 million to 5 million people in the United States. The deficiency may lead to soft tissue bruising, mucosal bleeding and fatal intracranial bleeding. Newborns with Factor XIII deficiency may have umbilical cord bleeding. "This product helps fill an important need," said Karen Midthun, M.D., director of the FDA's Center for Biologics ... Read more
Related support groups: Bleeding Disorder, Bleeding Associated with Coagulation Defect
Gene Therapy Corrected Rare Bleeding Disorder: Study
Posted 6 Dec 2010 by Drugs.com
SUNDAY, Dec. 5 – Using gene therapy, German researchers report that they managed to "correct" a malfunctioning gene responsible for Wiskott-Aldrich syndrome, a rare but devastating childhood disorder that leads to prolonged bleeding from even minor hits or scrapes, and also leaves these children vulnerable to certain cancers and dangerous infections. However, one of the 10 kids in the study developed acute T-cell leukemia, apparently as a result of the viral vector that was used to insert the healthy gene. The boy is currently on chemotherapy, the study authors noted. "This is a very good first step, but it's a little scary and we need to move to safer vectors," said Dr. Mary Ellen Conley, director of the Program in Genetic Immunodeficiencies at St. Jude Children's Research Hospital in Memphis, Tenn. "The study shows proof-of-principle that gene therapy with stem cells in a genetic ... Read more
Related support groups: Bleeding Disorder
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