A novel gene therapy for hemophilia A led to sustained expression of the clotting factor those patients lack, resulting in a reduction—or in some cases complete elimination—of painful and potentially life-threatening ...
Nov 17, 2021
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About 30 per cent of all women report heavy menstrual periods at some point during their reproductive years. Up to 15 per cent of these have an underlying bleeding disorder and yet most have never been diagnosed, leaving ...
Apr 15, 2019
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A Rush University Medical Center led international research team has announced that a treatment to prevent bleeding episodes in children with hemophilia A also is effective for adolescents and adults.
Jan 9, 2012
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The U.S. Food and Drug Administration on Thursday approved a costly single-dose gene therapy for patients with severe hemophilia A, a life-threatening hereditary bleeding disorder.
Jul 3, 2023
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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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For the first time, researchers have combined gene therapy and stem cell transplantation to successfully reverse the severe, crippling bleeding disorder hemophilia A in large animals, opening the door to the development of ...
Nov 3, 2011
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(Medical Xpress) -- An experimental gene therapy technique boosted the production of a vital blood clotting factor in six people with hemophilia B, according to new research supported by the National Institutes of Health. ...
Dec 13, 2011
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Research led by Randal J. Kaufman, Ph.D., has found that misfolded proteins in liver cells contribute to the development of liver cancer, shedding new light on the mysterious origins of one of the world's deadliest diseases. ...
Dec 7, 2022
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Alnylam Pharmaceuticals, Inc. announced today the publication in Nature Medicine of pre-clinical results with ALN-AT3, an investigational RNAi therapeutic targeting antithrombin (AT) for the treatment of hemophilia and rare ...
Apr 14, 2015
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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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