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Concizumab, sold under the brand name Alhemo, is a monoclonal antibody used for the treatment of hemophilia A and hemophilia B. [5] [8] It is an anti-tissue factor pathway inhibitor. [5] [8] The most common adverse reactions include injection site reactions and hives (urticaria). [11]
Efanesoctocog alfa is a recombinant DNA-derived, Factor VIII concentrate indicated for use in adults and children with hemophilia A (congenital factor VIII deficiency) for routine prophylaxis to reduce the frequency of bleeding episodes; on-demand treatment & control of bleeding episodes; and perioperative management of bleeding.
It is a single-dose treatment that gives the patient the genetic information required to produce Factor IX. [91] In June 2023, the FDA approved the first gene therapy treatment for haemophilia A, called Roctavian. It was only approved for patients with severe cases, but it has been shown to reduce yearly bleeding episodes by 50%.
Hemophilia is a family of rare genetic blood diseases caused by a clotting factor deficiency (FVIII in hemophilia A, FIX in hemophilia B), impacting more than 800,000 people globally.
My son battles severe factor 8 deficiency, commonly known as hemophilia, a chronic and life-threatening condition. Without regular infusions of factor, his blood fails to clot, putting him at ...
Contaminated hemophilia blood products were a serious public health problem in the late 1970s up to 1985. Hemophilia A causes a deficiency in Factor VIII , a protein required for blood clotting. Factor VIII injections are a common treatment to prevent or stop bleeding in people with hemophilia A. [ 1 ]
With the proven success of immunotherapy for cancer treatment, there is an emerging interest in utilizing the patient’s own immune system for cancer prevention, especially in high-risk ...
Etranacogene dezaparvovec, sold under the brand name Hemgenix is a gene therapy used for the treatment of hemophilia B. [5] [6] [7] Etranacogene dezaparvovec is an adeno-associated virus vector-based gene therapy which consists of a viral vector carrying a gene for clotting Factor IX. [7]