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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 ...
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]
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 ...
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 ...
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]
Its products include eculizumab (Soliris) and ravulizumab (Ultomiris), both used to treat the rare disorders of atypical hemolytic uremic syndrome (aHUS) and paroxysmal nocturnal hemoglobinuria (PNH); asfotase alfa (Strensiq), used to treat hypophosphatasia; sebelipase alfa (Kanuma), used to treat lysosomal acid lipase deficiency, and andexanet ...
Paroxysmal nocturnal hemoglobinuria (blood test) Viral infections (such as HIV, EBV; an undetermined virus is most common) Alimentary toxic aleukia; Copper deficiency; Pernicious anemia; Medication; Hypersplenism; Osteopetrosis; Organic acidurias (Propionic Acidemia, Methylmalonic Aciduria, Isovaleric Aciduria) Low dose arsenic poisoning
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.