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Danicopan, sold under the brand name Voydeya, is a medication used for the treatment of paroxysmal nocturnal hemoglobinuria. It is a complement inhibitor which reversibly binds to factor D to prevent alternative pathway-mediated hemolysis and deposition of complement C3 proteins on red blood cells. The most common side effects include fever, headache, increased levels of liver enzymes (a sign ...
Hemoglobinuria is a condition in which the oxygen transport protein hemoglobin is found in abnormally high concentrations in the urine. [1] The condition is caused by excessive intravascular hemolysis , in which large numbers of red blood cells (RBCs) are destroyed, thereby releasing free hemoglobin into the plasma . [ 2 ]
Ravulizumab, sold under the brand name Ultomiris, is a humanized monoclonal antibody complement inhibitor medication designed for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome. It is designed to bind to and prevent the activation of Complement component 5 (C5). [8] [7]
The sucrose lysis test is a diagnostic laboratory test used for diagnosing paroxysmal nocturnal hemoglobinuria (PNH), as well as for hypoplastic anemias and any hemolytic anemia with an unclear cause. [1] The test works by using sucrose, which creates a low ionic strength environment that allows complement to bind to red blood cells. [1]
Iptacopan, sold under the brand name Fabhalta, is a medication used for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). [4] It is a complement factor B inhibitor that was developed by Novartis. [4] It is taken by mouth. [4]
The European Medicines Agency approved it for the treatment of paroxysmal nocturnal hemoglobinuria in June 2007, [7] and in November 2011, for the treatment of atypical hemolytic uremic syndrome. [24] Health Canada approved it in 2009, to treat paroxysmal nocturnal hemoglobinuria and in 2013, as the only drug to treat atypical hemolytic uremic ...
COMMODORE 2 [14] is a phase III randomized trial comparing crovalimab vs eculizumab in people with paroxysmal nocturnal hemoglobinuria who are naive to C5 inhibitor treatment. [15] COMMODORE 2 was positive for its co-primary endpoints, transfusion avoidance and hemolysis control (measured lactate dehydrogenase levels) which are disease control ...
Mutations affecting GPI that reduce expression of CD59 and decay-accelerating factor on red blood cells result in paroxysmal nocturnal hemoglobinuria. [10] GPI mutation and consequent reduction in CD59 expression results from a cysteine to tyrosine missense mutation, which prevents disulfide bridge formation, ultimately disrupting tertiary ...