ALLMedicine™ Paroxysmal Nocturnal Hemoglobinuria Center

Jul 28th, 2021 - Paroxysmal nocturnal hemoglobinuria (PNH) is characterised by uncontrolled terminal complement activation and subsequent intravascular hemolysis (IVH). C5 inhibitors prevent membrane attack complex formation, but patients may experience extravascu...

Jul 19th, 2021 - To evaluate the economic burden and treatment patterns of patients with paroxysmal nocturnal hemoglobinuria (PNH) treated with eculizumab, a C5 inhibitor, who were defined as blood transfusion-dependent (TD) versus blood transfusion-free (TF) in t...

Jul 17th, 2021 - The presence of paroxysmal nocturnal hemoglobinuria (PNH) clones in aplastic anemia (AA) suggests immunopathogenesis, but when and how PNH clones emerge and proliferate are unclear. Hepatitis-associated aplastic anemia (HAAA) is a special variant ...

Jul 10th, 2021 - Paroxysmal nocturnal hemoglobinuria (PNH) is a disease caused by a phosphatidylinositol glycan anchor biosynthesis class A (PIG-A) mutation in hematopoietic stem cells. There are three theories about the possible mechanism of the pathogenesis of P...

Jul 1st, 2021 - The complement system is an important defense mechanism against pathogens; however, in certain pathologies, the system also attacks human cells, such as red blood cells (RBCs). In paroxysmal nocturnal hemoglobinuria (PNH), RBCs lack certain comple...

Jun 10th, 2010 - Paroxysmal nocturnal hemoglobinuria (PNH) is a rare hematopoietic stem cell disorder characterized by a somatic mutation in the PIGA gene, leading to a deficiency of proteins linked to the cell membrane via glycophosphatidylinositol (GPI) anchors....

Apr 7th, 2020 - Patients with malignant and non-malignant hematologic diseases including severe aplastic anemia (SAA), paroxysmal nocturnal hemoglobinuria (PNH), myelodysplastic syndrome (MDS), acute and chronic leukemias, Hodgkin's and non-Hodgkin's lymphoma and...

Mar 2nd, 2020 - Paroxysmal nocturnal hemoglobinuria (PNH) is a rare acquired clonal hematological disorder leading to red blood cells hemolysis and thrombosis. PNH has been reported to be the cause of cerebral venous thrombosis and embolic ischemic strokes and is...

Nov 8th, 2019 - To determine whether LFG316 can induce a hematological response, as measured by reduction in hemolytic activity, in patients with PNH.

Jun 19th, 2019 - The purpose of this study is to enable continued access to zilucoplan (RA101495) for patients with paroxysmal nocturnal hemoglobinuria (PNH) after they complete a zilucoplan clinical study.

Jun 4th, 2019 - This study is ongoing. The data presented is up to the Primary Completion date of the study and is for the Primary Evaluation period.

May 19th, 2021 - A rare, life-threatening anemia now has a new treatment option. The Food and Drug Administration announced the approval of pegcetacoplan (Empaveli) injection to treat adults with paroxysmal nocturnal hemoglobinuria (PNH). Pegcetacoplan is the firs...

Dec 17th, 2020 - ZURICH (Reuters) - Novartis got the U.S. Food and Drug Administration's Breakthrough Therapy tag for its experimental medicine iptacopan, which the Swiss drugmaker hopes wins approval to treat the rare blood disorder paroxysmal nocturnal hemoglobi...

Apr 20th, 2020 - (Reuters) - Alexion Pharmaceuticals Inc said on Monday it would start a late-stage study of its rare blood-disorder drug, Ultomiris, in COVID-19 patients hospitalized with severe pneumonia or acute respiratory distress syndrome. The study is expec...

Jan 21st, 2020 - The introduction of eculizumab, a monoclonal antibody targeting C5 of the complement cascade, revolutionized the treatment of paroxysmal nocturnal hemoglobinuria (PNH), a rare hematologic disorder characterized by complement-mediated intravascular.

Oct 21st, 2019 - The US Food and Drug Administration (FDA) has approved ravulizumab (Ultomiris, Alexion Pharmaceuticals) to inhibit complement-mediated thrombotic microangiopathy (TMA) in adults and children age 1 month or older with atypical hemolytic uremic synd...