US approves gene therapy treatment for hemophilia
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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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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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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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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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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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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Haemophilia ( /hiːməˈfɪliə/; also spelled hemophilia in North America, from the Greek haima αἷμα 'blood' and philia φιλος 'love') is a group of hereditary genetic disorders that impair the body's ability to control blood clotting or coagulation, which is used to stop bleeding when a blood vessel is broken. Haemophilia A (clotting factor VIII deficiency) is the most common form of the disorder, present in about 1 in 5,000–10,000 male births. Haemophilia B (factor IX deficiency) occurs in around 1 in about 20,000–34,000 male births.
Like most recessive sex-linked, X chromosome disorders, haemophilia is more likely to occur in males than females. This is because females have two X chromosomes while males have only one, so the defective gene is guaranteed to manifest in any male who carries it. Because females have two X chromosomes and haemophilia is rare, the chance of a female having two defective copies of the gene is very remote, so females are almost exclusively asymptomatic carriers of the disorder. Female carriers can inherit the defective gene from either their mother or father, or it may be a new mutation. Although it is not impossible for a female to have haemophilia, it is unusual: a female with haemophilia A or B would have to be the daughter of both a male haemophiliac and a female carrier, while the non-sex-linked haemophilia C due to coagulant factor XI deficiency, which can affect either sex, is more common in Jews of Ashkenazi (east European) descent but rare in other population groups.
Haemophilia lowers blood plasma clotting factor levels of the coagulation factors needed for a normal clotting process. Thus when a blood vessel is injured, a temporary scab does form, but the missing coagulation factors prevent fibrin formation, which is necessary to maintain the blood clot. A haemophiliac does not bleed more intensely than a person without it, but can bleed for a much longer time. In severe haemophiliacs even a minor injury can result in blood loss lasting days or weeks, or even never healing completely. In areas such as the brain or inside joints, this can be fatal or permanently debilitating.
This text uses material from Wikipedia licensed under CC BY-SA