In a study published today in the journal Nature Genetics, a group of scientists including UNC biologist Jason Lieb, PhD, present experiments supporting a longstanding hypothesis that explains how males can survive with only ...
Oct 24, 2011
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A genetically engineered clotting factor that controlled hemophilia in an animal study offers a novel potential treatment for human hemophilia and a broad range of other bleeding problems.
Oct 23, 2011
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Patients with hemophilia A or B with inhibitors have a lower annualized bleeding rate with concizumab than with no prophylaxis, according to a phase 3 study published online Aug. 31 in the New England Journal of Medicine.
Sep 3, 2023
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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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People with one form of the genetic blood disorder hemophilia now have a one-time treatment with a $3.5 million price tag.
Nov 23, 2022
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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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Researchers at Children's Hospital of Philadelphia (CHOP) have identified a key target that may be responsible for treatment failure in about 30% of patients with hemophilia A. The target, known as B cell activating factor ...
Apr 15, 2021
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In a new study from the Wake Forest Institute for Regenerative Medicine (WFIRM) researchers have developed an optimized cellular platform for delivering Factor 8 to better treat patients with hemophilia A.
Jul 28, 2020
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Children with severe hemophilia A who receive early prophylaxis are less likely to have joint damage in young adulthood than those who begin treatment later, according to a new study in Blood Advances. While data suggest ...
Jun 3, 2020
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Children (and adults) with hemophilia are slow to form blood clots, so are at constant risk for uncontrolled bleeding. Even when the skin isn't broken, a fall or a simple toe stub can become a serious medical issue: internal ...
Mar 10, 2020
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