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Factor VIII is a medication used to treat and prevent bleeding in people with hemophilia A and other causes of low factor VIII. [12] [13] Certain preparations may also be used in those with von Willebrand's disease. [13] It is given by slow injection into a vein. [12]
This list of over 500 monoclonal antibodies includes approved and investigational drugs as well as drugs that have been withdrawn from market; consequently, the column Use does not necessarily indicate clinical usage. See the list of FDA-approved therapeutic monoclonal antibodies in the monoclonal antibody therapy page.
Hemophilia [7] Von Willebrand disease [8] Joint damage, potentially with severe pain and even destruction of the joint and development of arthritis: Hemophilia [7] Von Willebrand disease [8] Retinal bleeding: Acute leukemia [5] Transfusion transmitted infection, from blood transfusions that are given as treatment. Hemophilia [7]
Lymphedema is most frequently a complication of cancer treatment or parasitic infections, but it can also be seen in a number of genetic disorders. Tissues with lymphedema are at high risk of infection because the lymphatic system has been compromised. [3] Though incurable and progressive, a number of treatments may improve symptoms. [2]
Coagulopathy (also called a bleeding disorder) is a condition in which the blood's ability to coagulate (form clots) is impaired. [1] This condition can cause a tendency toward prolonged or excessive bleeding (bleeding diathesis), which may occur spontaneously or following an injury or medical and dental procedures.
Up to 20% of people develop antibodies to the clotting factors which makes treatment more difficult. [3] The medication desmopressin may be used in those with mild haemophilia A. [16] Studies of gene therapy are in early human trials. [17] Haemophilia A affects about 1 in 5,000–10,000, while haemophilia B affects about 1 in 40,000 males at birth.
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]
Delandistrogene moxeparvovec (Elevidys): treatment for Duchenne muscular dystrophy [7] [8] Elivaldogene autotemcel (Skysona): treatment for cerebral adrenoleukodystrophy [9] Etranacogene dezaparvovec (Hemgenix): AAV-based treatment for hemophilia B [10] Exagamglogene autotemcel (Casgevy): treatment for sickle cell disease. [11]
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