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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 ...
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 ...
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 ]
Symptoms of AIHA may be due to the underlying anemia; including shortness of breath or dyspnea, fatigue, headache, muscle weakness and pallor. [10] In cold agglutinin disease (cold antibody type), agglutination and impaired passage of red blood cells through capillaries in the extremities causes acrocyanosis and Raynaud phenomenon with a rare complication of gangrene [4]
CHAPLE syndrome is characterized by complement-mediated autoimmune hemolysis and paroxysmal nocturnal hemoglobinuria.The protein CD55 (also called decay-accelerating factor) helps to regulate the complement cascade, part of the innate immune system, by regulating the amplification phase.
Paroxysmal cold hemoglobinuria [2] Drug-induced autoimmune hemolytic anemia [2] Alloimmune hemolytic anemia [1] Hemolytic disease of the newborn (HDN) [1] Rh disease (Rh D) [1] ABO hemolytic disease of the newborn [1] Anti-Kell hemolytic disease of the newborn [1] Rhesus c hemolytic disease of the newborn [1] Rhesus E hemolytic disease of the ...
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]
Paroxysmal nocturnal hemoglobinuria, an acquired hematologic disorder, has been shown to result from somatic mutations in this gene. Alternate splice variants have been characterized. Alternate splice variants have been characterized.