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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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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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 single gene therapy injection could dramatically reduce the bleeding risk faced by people with hemophilia B, finds a study involving UCL researchers.
Jul 20, 2022
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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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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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Gene therapy researchers have produced a bioengineered decoy that fools the immune system and prevents it from mistakenly defeating the benefits delivered by a corrective gene. The decoy was effective in animal studies, and ...
Jul 17, 2013
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(HealthDay)—Rixubis (coagulation factor IX recombinant) has been approved by the U.S. Food and Drug Administration to prevent routine bleeding episodes in people aged 16 and older with hemophilia B, the agency said in a ...
Jun 28, 2013
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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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(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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