An international team of researchers has found more evidence that a new gene therapy may help some people with hemophilia A. In their paper published in the New England Journal of Medicine, the group describes the characteristics ...
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 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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For nearly a third of patients with hemophilia A and almost all patients with Pompe disease, their own immune system is their greatest obstacle to treatment. When given essential proteins and enzymes, their body perceives ...
Sep 30, 2021
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A St. Jude Children's Research Hospital analysis found a major gap between the cost to manufacture and distribute hemophilia B gene therapy and the $2 million-plus price reportedly under consideration for hemophilia gene ...
Jun 4, 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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Within the framework of an international study, Lund University and Skåne University Hospital have started treating patients with hemophilia with gene therapy, something that began in January this year. The hope is that ...
Jan 29, 2020
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