MeSH tree: C15.378.463.510
For men with hemophilia B, receipt of gene therapy comprising an infusion of etranacogene dezaparvovec results in sustained endogenous factor IX expression and low annualized bleeding rates over five years, according to a ...
Dec 10, 2025
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After blood vessel damage, effective blood clotting is essential to halt bleeding. However, this process is inefficient in some individuals due to hereditary factors. Hemophilia B, for example, results from a deficiency in ...
Sep 29, 2025
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A gene transfer approach to treating the bleeding disorder hemophilia B remains safe and effective long-term, as scientists from St. Jude Children's Research Hospital and University College London today report thirteen years ...
Jun 12, 2025
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The U.S. Food and Drug Administration has approved Sanofi's Qfitlia (fitusiran) for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with ...
Apr 3, 2025
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The U.S. Food and Drug Administration (FDA) approved Hympavzi (marstacimab-hncq) for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients aged 12 years and older with ...
Oct 15, 2024
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Adults with hemophilia B saw their number of bleeding episodes drop by an average of 71% after a single infusion of gene therapy, according to the results of an international Phase III clinical trial published in the New ...
Sep 25, 2024
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61
In a clinical practice guideline issued by the International Society on Thrombosis and Haemostasis and published online June 13 in the Journal of Thrombosis and Haemostasis, recommendations are presented for the treatment ...
Jul 1, 2024
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Pharmaceutical giant Pfizer has received US approval for a gene therapy against a form of hemophilia, a rare and inherited blood clotting disorder, the company said Friday.
Apr 26, 2024
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For patients with hemophilia B, one infusion of adeno-associated virus 5 (AAV5) vector expressing the Padua factor IX variant (etranacogene dezaparvovec) is noninferior and superior to prophylaxis with factor IX, according ...
Mar 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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