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About 2,037 results

ALLMedicine™ Paroxysmal Nocturnal Hemoglobinuria Center

Research & Reviews  963 results

C3 inhibition with pegcetacoplan in subjects with paroxysmal nocturnal hemoglobinuria t...
https://doi.org/10.1002/ajh.25960
American Journal of Hematology; de Castro C, Grossi F et. al.

Jan 19th, 2021 - Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired, life-threatening hematologic disease characterized by chronic complement-mediated hemolysis and thrombosis. Despite treatment with eculizumab, a C5 inhibitor, 72% of individuals remain anem...

Dental infection associated with exuberant gingival necrosis in a patient with paroxysm...
https://doi.org/10.1111/scd.12552
Special Care in Dentistry : Official Publication of the A... Ximenes IDS, Filho OVO et. al.

Dec 28th, 2020 - Paroxysmal nocturnal hemoglobinuria (PNH) is a hematological disorder that affects hematopoietic stem cells. An association with other hematological diseases, such as hemolytic anemia and neutropenia, is observed with a high occurrence of aplastic...

Ultradeep Sequencing Analysis of Paroxysmal Nocturnal Hemoglobinuria Clones Detected by...
https://doi.org/10.1093/ajcp/aqaa211
American Journal of Clinical Pathology; Jeong D, Park HS et. al.

Dec 21st, 2020 - We aimed to determine whether small paroxysmal nocturnal hemoglobinuria (PNH) clones detected by flow cytometry (FCM) harbor PIG gene mutations with quantitative correlation. We analyzed 89 specimens from 63 patients whose PNH clone size was ≥0.1%...

Frequency and perturbations of various peripheral blood cell populations before and aft...
https://doi.org/10.1016/j.bcmd.2020.102528
Blood Cells, Molecules & Diseases; Gurnari C, Graham AC et. al.

Dec 20th, 2020 - While red blood cells (RBCs) and granulocytes have been more studied, platelets and reticulocytes are not commonly used in paroxysmal nocturnal hemoglobinuria (PNH) flow-cytometry and less is known about susceptibility to complement-mediated destr...

One-year outcomes from a phase 3 randomized trial of ravulizumab in adults with paroxys...
https://doi.org/10.1111/ejh.13564
European Journal of Haematology REFERENCES; Kulasekararaj AG, Hill A et. al.

Dec 10th, 2020 - Ravulizumab every 8 weeks showed non-inferiority to eculizumab every 2 weeks in a 26-week, phase 3, randomized controlled trial in adults with paroxysmal nocturnal hemoglobinuria (PNH) who were clinically stable on eculizumab (NCT03056040). We rep...

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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  1,001 results

C3 inhibition with pegcetacoplan in subjects with paroxysmal nocturnal hemoglobinuria t...
https://doi.org/10.1002/ajh.25960
American Journal of Hematology; de Castro C, Grossi F et. al.

Jan 19th, 2021 - Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired, life-threatening hematologic disease characterized by chronic complement-mediated hemolysis and thrombosis. Despite treatment with eculizumab, a C5 inhibitor, 72% of individuals remain anem...

Dental infection associated with exuberant gingival necrosis in a patient with paroxysm...
https://doi.org/10.1111/scd.12552
Special Care in Dentistry : Official Publication of the A... Ximenes IDS, Filho OVO et. al.

Dec 28th, 2020 - Paroxysmal nocturnal hemoglobinuria (PNH) is a hematological disorder that affects hematopoietic stem cells. An association with other hematological diseases, such as hemolytic anemia and neutropenia, is observed with a high occurrence of aplastic...

Ultradeep Sequencing Analysis of Paroxysmal Nocturnal Hemoglobinuria Clones Detected by...
https://doi.org/10.1093/ajcp/aqaa211
American Journal of Clinical Pathology; Jeong D, Park HS et. al.

Dec 21st, 2020 - We aimed to determine whether small paroxysmal nocturnal hemoglobinuria (PNH) clones detected by flow cytometry (FCM) harbor PIG gene mutations with quantitative correlation. We analyzed 89 specimens from 63 patients whose PNH clone size was ≥0.1%...

Frequency and perturbations of various peripheral blood cell populations before and aft...
https://doi.org/10.1016/j.bcmd.2020.102528
Blood Cells, Molecules & Diseases; Gurnari C, Graham AC et. al.

Dec 20th, 2020 - While red blood cells (RBCs) and granulocytes have been more studied, platelets and reticulocytes are not commonly used in paroxysmal nocturnal hemoglobinuria (PNH) flow-cytometry and less is known about susceptibility to complement-mediated destr...

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...

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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 →