MeSH tree: C15.378.100.100
Patients with the genetic disorder hemophilia A receive factor VIII protein replacement treatments to replenish this clotting protein in their blood, thus preventing dangerous bleeding. Unfortunately, about 30% of these patients ...
Aug 14, 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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Catherine Reitzel's multiple sclerosis medication costs nearly $100,000 a year. Kris Garcia relies on a drug for a blood-clotting disorder that runs $10,000 for a three-day supply. And Mariana Marquez-Farmer would likely ...
May 31, 2023
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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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For patients with hemophilia A, factor VIII activity and bleeding reduction persist at two years after gene transfer with valoctocogene roxaparvovec, which delivers a B-domain-deleted factor VIII coding sequence with an adeno-associated ...
Mar 3, 2023
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In hemophilia A cases, the body either partially or completely lacks the blood coagulation factor VIII (FVIII), or the factor is formed incorrectly. Patients usually receive FVIII that comes from donor blood or is produced ...
Feb 7, 2023
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A global study involving McMaster University researchers has trialed a potential new treatment that could be a game-changer for people living with hemophilia A, a life-threatening genetic blood disorder.
Jan 26, 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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Immune responses against Factor VIII, which is used to treat hemophilia A (bleeding disorder), are influenced by the microenvironment of antigen presenting cells. This is the result of a scientific study conducted in cooperation ...
Nov 22, 2022
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