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About 1,115 results

ALLMedicine™ Paroxysmal Nocturnal Hemoglobinuria Center

Research & Reviews  370 results

Phase 2 Study of Danicopan in Paroxysmal Nocturnal Hemoglobinuria Patients with an Inad...
https://doi.org/10.1182/blood.2021011388
Blood Kulesekararaj A, Risitano AM et. al.

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...

Advances in the creation of animal models of paroxysmal nocturnal hemoglobinuria.
https://doi.org/10.1080/16078454.2021.1945244
Hematology (Amsterdam, Netherlands); Chen Y, Rong F

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...

Halting targeted and collateral damage to red blood cells by the complement system.
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8243056
Seminars in Immunopathology; Jalink M, de Boer ECW et. al.

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...

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Guidelines  1 results

Guidelines for the diagnosis and monitoring of paroxysmal nocturnal hemoglobinuria and ...
https://doi.org/10.1002/cyto.b.20525
Cytometry. Part B, Clinical Cytometry; Borowitz MJ, Craig FE et. al.

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....

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Drugs  6 results see all →

Clinicaltrials.gov  21 results

Specialized Blood Cell Transplants for Cancers of the Blood and Bone Marrow
https://clinicaltrials.gov/ct2/show/NCT00003838

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...

Paroxysmal Nocturnal Hemoglobinuria in ESUS & ETUS
https://clinicaltrials.gov/ct2/show/NCT03329365

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...

Proof of Concept Study to Assess the Efficacy, Safety and Pharmacokinetics of LFG316 in Patients With Paroxysmal Nocturnal Hemoglobinuria
https://clinicaltrials.gov/ct2/show/NCT02534909

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

Extension Study of RA101495 for Patients With PNH Who Have Completed a Zilucoplan (RA101495) Clinical Study
https://clinicaltrials.gov/ct2/show/NCT03225287

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.

Dose-Escalation Study of ALXN1210 IV in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
https://clinicaltrials.gov/ct2/show/NCT02598583

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.

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News  47 results

FDA Approves New Treatment Option for Rare Anemia
https://www.medscape.com/viewarticle/951476

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...

Novartis Gets FDA Breakthrough Therapy Tag for Experimental Iptacopan
https://www.medscape.com/viewarticle/942735

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...

Alexion to study rare blood-disorder drug in severe COVID-19 patients
https://www.reuters.com/article/us-health-coronavirus-alexion-pharms/alexion-to-study-rare-blood-disorder-drug-in-severe-covid-19-patients-idUSKBN2221PM

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...

The evolving landscape of complement inhibition therapy
https://www.mdedge.com/fedprac/article/215831/anemia/evolving-landscape-complement-inhibition-therapy?channel=41022
I. Romina Sosa, MD, PhD

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.

FDA Clears Ravulizumab for Atypical Hemolytic Uremic Syndrome
https://www.medscape.com/viewarticle/920120

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...

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Patient Education  1 results see all →