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About 2,057 results

ALLMedicine™ Paroxysmal Nocturnal Hemoglobinuria Center

Research & Reviews  973 results

Pegcetacoplan versus Eculizumab in Paroxysmal Nocturnal Hemoglobinuria.
https://doi.org/10.1056/NEJMoa2029073
The New England Journal of Medicine; Hillmen P, Szer J et. al.

Mar 17th, 2021 - Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired disease characterized by chronic complement-mediated hemolysis. C5 inhibition controls intravascular hemolysis in untreated PNH but cannot address extravascular hemolysis. Pegcetacoplan...

Risk of Disseminated Gonococcal Infections With Terminal Complement Blockade.
https://doi.org/10.1097/MPH.0000000000002075
Journal of Pediatric Hematology/oncology; Graciaa SH, Graciaa DS et. al.

Feb 9th, 2021 - Paroxysmal nocturnal hemoglobinuria is a clonal hematopoietic stem cell disorder resulting in complement-mediated hemolysis. Eculizumab, a monoclonal antibody against complement protein C5, has been shown to reduce both intravascular hemolysis and...

Key Points for Issue.
https://doi.org/10.1182/blood-2012-07-441857
Continuum (Minneapolis, Minn.);

Feb 1st, 2021 - CD59 deficiency is a common finding in RBCs and WBCs in patients with chronic hemolysis suffering from paroxysmal nocturnal hemoglobinuria in which the acquired mutation in the PIGA gene leads to membrane loss of glycosylphosphatidylinositol-ancho...

How I treat paroxysmal nocturnal hemoglobinuria.
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7955407
Blood Brodsky RA

Jan 29th, 2021 - Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, clonal, complement-mediated hemolytic anemia with protean manifestations. PNH can present as a hemolytic anemia, a form of bone marrow failure, a thrombophilia, or any combination of the above. ...

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Guidelines  2 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 9th, 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....

SOLIRIS® (ECULIZUMAB)
https://alexion.com/Products/Soliris

Soliris® is a first-in-class terminal complement inhibitor discovered, developed, and commercialized by Alexion. Soliris® works by inhibiting terminal complement, a part of the immune system that, when activated in an uncontrolled manner, plays a...

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Drugs  5 results see all →

Clinicaltrials.gov  1,011 results

Ultomiris - ravulizumab solution, concentrate-Alexion Pharmaceuticals Inc.
https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=a9a590d9-0217-43c7-908d-e62a71279791

Apr 5th, 2021 - ULTOMIRIS is indicated for: the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH). the treatment of adults and pediatric patients one month of age and older with atypical hemolytic uremic syndrome (aHUS) to inhibit complem...

Pegcetacoplan versus Eculizumab in Paroxysmal Nocturnal Hemoglobinuria.
https://doi.org/10.1056/NEJMoa2029073
The New England Journal of Medicine; Hillmen P, Szer J et. al.

Mar 17th, 2021 - Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired disease characterized by chronic complement-mediated hemolysis. C5 inhibition controls intravascular hemolysis in untreated PNH but cannot address extravascular hemolysis. Pegcetacoplan...

Risk of Disseminated Gonococcal Infections With Terminal Complement Blockade.
https://doi.org/10.1097/MPH.0000000000002075
Journal of Pediatric Hematology/oncology; Graciaa SH, Graciaa DS et. al.

Feb 9th, 2021 - Paroxysmal nocturnal hemoglobinuria is a clonal hematopoietic stem cell disorder resulting in complement-mediated hemolysis. Eculizumab, a monoclonal antibody against complement protein C5, has been shown to reduce both intravascular hemolysis and...

Key Points for Issue.
https://doi.org/10.1182/blood-2012-07-441857
Continuum (Minneapolis, Minn.);

Feb 1st, 2021 - CD59 deficiency is a common finding in RBCs and WBCs in patients with chronic hemolysis suffering from paroxysmal nocturnal hemoglobinuria in which the acquired mutation in the PIGA gene leads to membrane loss of glycosylphosphatidylinositol-ancho...

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News  65 results

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

Dec 16th, 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...

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 20th, 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...

C3 inhibitor shows potential in PNH and AIHA
https://www.mdedge.com/hematology-oncology/article/199135/anemia/c3-inhibitor-shows-potential-pnh-and-aiha?channel=39313
Will Pass

Apr 18th, 2019 - GLASGOW – APL-2, a complement factor 3 (C3) inhibitor, may be a future treatment option for paroxysmal nocturnal hemoglobinuria (PNH) and autoimmune hemolytic anemia (AIHA), according to investigators from two separate studies. Will Pass/MDedge Ne.

FDA approves ravulizumab for PNH
https://www.mdedge.com/hematology-oncology/article/192205/anemia/fda-approves-ravulizumab-pnh
HT Staff

Dec 25th, 2018 - Photo from Business Wire Ravulizumab (Ultomiris) The U. S.

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Patient Education  1 results see all →