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Discovering C3 targeting therapies for paroxysmal nocturnal hemoglobinuria: Achievements and pitfalls.

Seminars in Immunology, 2022
The treatment of paroxysmal nocturnal hemoglobinuria (PNH) was revolutionized by the introduction of the anti-C5 agent eculizumab, which resulted in sustained control of intravascular hemolysis, leading to transfusion avoidance and hemoglobin ...
A. Risitano   +3 more
semanticscholar   +1 more source

Factor B inhibitor iptacopan for the treatment of paroxysmal nocturnal hemoglobinuria.

Blood reviews
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, clonal, complement-mediated hemolytic anemia with a variety of manifestations. Currently, the methods for treating PNH include anti-C5 treatments (eculizumab and ravulizumab) and pegcetacoplan (a ...
Bo Xu   +4 more
semanticscholar   +1 more source

Paroxysmal Nocturnal Hemoglobinuria

Mayo Clinic Proceedings
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired clonal hematologic disorder characterized by intravascular destruction of red blood cells by the complement system. This intravascular hemolysis can lead to a constellation of symptoms, including anemia, fatigue, shortness of breath, smooth muscle dystonia, and dark urine that is classically
Richard C. Godby, Surbhi Shah
openaire   +2 more sources

Pregnancy and Paroxysmal Nocturnal Hemoglobinuria

Archives of Internal Medicine, 1988
Our study concerns eight pregnancies, six of which were successful, in four patients with paroxysmal nocturnal hemoglobinuria (PNH). Several complications of PNH during pregnancy were prevented: chronic anemia, folate and iron deficiency, and deep-vein thrombosis.
P, Solal-Céligny   +8 more
openaire   +2 more sources

Paroxysmal Nocturnal Hemoglobinuria in Children

Pediatric Drugs, 2007
Paroxysmal nocturnal hemoglobinuria (PNH), an acquired hematologic disorder characterized by intravascular hemolysis, nocturnal hemoglobinuria, thrombotic events, serious infections, and bone marrow failure, is very rare in children. PNH is caused by a somatic mutation of the phosphatidylinositol glycan (GPI) complementation class A (PIGA) gene ...
openaire   +3 more sources

Pregnancy and paroxysmal nocturnal hemoglobinuria

European Journal of Obstetrics & Gynecology and Reproductive Biology, 1994
A patient is described who developed symptoms of paroxysmal nocturnal hemoglobinuria (PNH) in her first pregnancy. This was uneventful except for a spontaneous preterm delivery. The second pregnancy was complicated by severe anemia and a hemolytic crisis with Budd-Chiari syndrome at 31 weeks' amenorrhoea.
Bais, J.   +3 more
openaire   +3 more sources

Paroxysmal Nocturnal Hemoglobinuria

New England Journal of Medicine, 1961
PAROXYSMAL nocturnal hemoglobinuria is a disease with a fascinating diversity of challenges and implications. These have prompted numerous clinical and basic investigations. The clinical features are often bizarre and may be misleading, even with the most typical presentation of abdominal pain, fatigue, weakness, anemia and dark urine and with venous ...
openaire   +2 more sources

Paroxysmal Nocturnal Hemoglobinuria

1992
Paroxysmal nocturnal hemoglobinuria (PNH) is a clonal disorder of the hematopoietic stem cell (Hartmann and Arnold 1977) resulting in the production of blood cells which are defective in that they lack or are markedly deficient in glycan-phosphatidylinositol (GPI)-linked surface proteins (Rosse 1990a).
openaire   +2 more sources

Paroxysmal Nocturnal Hemoglobinuria Revisited

New England Journal of Medicine, 1983
Immunologic systems are now known to have a major role in the pathophysiology of many human disease processes. One such system is serum complement.
openaire   +2 more sources

Paroxysmal nocturnal hemoglobinuria

Medicina clinica, 2009
Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired clonal hematopoietic disorder characterized by the existence of somatic mutations in the PIG-A (phosphatidylinositolglycan complementation class A) gene, which encodes for a protein involved in the biosynthesis of the glycosyl phosphatidylinositol (GPI) molecule that serves as an anchor for many ...
Pilar M, Hernández-Campo   +2 more
openaire   +3 more sources

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