(Medical Xpress) -- Scientists have used a gene therapy tool that acts like intelligent molecular scissors to correct the key gene defect in mice with hemophilia B, a disease that can lead to uncontrolled bleeding. The intervention ...
Jun 28, 2011
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A razor nick in the shower? A fall on a slippery floor? The average person simply calls it bad karma, but for people with hemophilia such episodes can cause prolonged blood loss.
Jun 27, 2011
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Researchers determined the minimally effective dose (MED) of a clinical candidate gene therapy vector for treating hemophilia A. This pharmacology study, performed in a mouse model and designed to support the initiation of ...
Dec 16, 2021
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A new study examined 42 combinations of promoters and enhancers for human factor VIII (hFVIII) gene expression to identify the optimal adeno-associated virus (AAV)-based gene therapy delivery vector constructs to take forward ...
Mar 31, 2017
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The manner in which pharmaceutical companies market their products to people who have hemophilia appears unprecedented and direct-to-consumer marketing should be examined by regulators, say researchers who reviewed documents, ...
Jun 14, 2016
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The drug Alprolix has been approved by the U.S. Food and Drug Administration as the first long-acting hemophilia B clotting agent, the FDA said in a news release.
Mar 31, 2014
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Prothrombin complex concentrates (PCCs) are faster and more effective than fresh frozen plasma at reversing hemorrhage caused by the anti-coagulant warfarin, despite plasma being the most commonly used therapy. A literature ...
Aug 2, 2013
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The time for commercial development of gene therapy has come. Patients with diseases treatable and curable with gene therapy deserve access to the technology, which has demonstrated both its effectiveness and feasibility, ...
Jan 24, 2012
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An international research team led by Dr. Cindy Leissinger of Tulane University School of Medicine, along with Dr. Alessandro Gringeri from the University of Milan, has found that a drug commonly used to treat bleeding events ...
Nov 2, 2011
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The Bleeding Disease: Hemophilia and the Unintended Consequences of Medical Progress (John Hopkins University Press) is the new book by NJIT Associate Professor Stephen Pemberton. The book recounts the promising and perilous ...
Aug 18, 2011
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