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About 1,723 results

ALLMedicine™ Paroxysmal Nocturnal Hemoglobinuria Center

Research & Reviews  523 results

Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis
https://clinicaltrials.gov/ct2/show/NCT03520647

Jul 1st, 2022 - Severe aplastic anemia (SAA),myelodysplastic syndrome (MDS), and paroxysmal nocturnal hemoglobinuria (PNH) are life-threatening bone marrow disorders. For SAA patients, long term survival can be achieved with immunosuppressive treatment. However, ...

Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused W...
https://clinicaltrials.gov/ct2/show/NCT01174108

Jul 1st, 2022 - Allogeneic hematopoietic stem cell transplantation (aHSCT) can cure patients with a variety of bone marrow failure syndromes (BMFS) including severe aplastic anemia (SAA), paroxysmal nocturnal hemoglobinuria (PNH) or myelodysplastic syndrome (MDS)...

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

Jul 1st, 2022 - 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...

Discovering C3 targeting therapies for paroxysmal nocturnal hemoglobinuria: Achievement...
https://doi.org/10.1016/j.smim.2022.101618
Seminars in Immunology; Risitano AM, Peffault de Latour R et. al.

Jun 29th, 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 stab...

A Long-Term Treatment Study of ACH-0144471 in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
https://clinicaltrials.gov/ct2/show/NCT03181633

Jun 28th, 2022 - The purpose of this study is to evaluate the long-term safety and efficacy of ACH-0144471 in participants with paroxysmal nocturnal hemoglobinuria (PNH) who have demonstrated clinical benefit from ACH-0144471 in Study ACH471-100. This study is des...

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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  84 results

Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis
https://clinicaltrials.gov/ct2/show/NCT03520647

Jul 1st, 2022 - Severe aplastic anemia (SAA),myelodysplastic syndrome (MDS), and paroxysmal nocturnal hemoglobinuria (PNH) are life-threatening bone marrow disorders. For SAA patients, long term survival can be achieved with immunosuppressive treatment. However, ...

Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused W...
https://clinicaltrials.gov/ct2/show/NCT01174108

Jul 1st, 2022 - Allogeneic hematopoietic stem cell transplantation (aHSCT) can cure patients with a variety of bone marrow failure syndromes (BMFS) including severe aplastic anemia (SAA), paroxysmal nocturnal hemoglobinuria (PNH) or myelodysplastic syndrome (MDS)...

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

Jul 1st, 2022 - 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...

A Long-Term Treatment Study of ACH-0144471 in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
https://clinicaltrials.gov/ct2/show/NCT03181633

Jun 28th, 2022 - The purpose of this study is to evaluate the long-term safety and efficacy of ACH-0144471 in participants with paroxysmal nocturnal hemoglobinuria (PNH) who have demonstrated clinical benefit from ACH-0144471 in Study ACH471-100. This study is des...

Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH)(ALPHA)
https://clinicaltrials.gov/ct2/show/NCT04469465

Jun 23rd, 2022 - This is a multiple-region, randomized, double-blind, placebo controlled, multiple-dose, study in participants with PNH who have clinically evident EVH on a C5 inhibitor (eculizumab or ravulizumab). Participants will be randomized to receive danico...

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News  73 results

Pegcetacoplan Improves Efficacy in Complement Inhibitor–Naïve Paroxysmal Nocturnal Hemoglobinuria
https://www.onclive.com/view/pegcetacoplan-improves-efficacy-in-complement-inhibitor-na-ve-paroxysmal-nocturnal-hemoglobinuria

Apr 9th, 2022 - Pegcetacoplan (Empaveli) treatment was associated with hemoglobin stabilization and normalization, as well as a superior change from baseline in hemoglobin levels, which showed a meaningful correction of anemia, in patients with complement inhibit...

Phase 3 Drug Trial for ALS Halted for Lack of Efficacy
https://www.medscape.com/viewarticle/957004

Aug 20th, 2021 - A global phase 3 trial of ravulizumab (Ultomiris) for adults with amyotrophic lateral sclerosis (ALS) is being discontinued following a data review, its manufacturer has announced. The CHAMPION-ALS placebo-controlled trial had enrolled 382 adults ...

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...

FDA approves new treatment option for rare anemia
https://www.mdedge.com/hematology-oncology/article/240251/anemia/fda-approves-new-treatment-option-rare-anemia
Mark S. Lesney, PhD

May 18th, 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).

VEXAS Syndrome: Implications for Dermatologists
https://www.medscape.com/viewarticle/949785

Apr 22nd, 2021 - When I was a medical student, I always found it gratifying when there was a unifying mechanism that explained the symptoms of a disease. Part of the reason I chose dermatology as a specialty was how frequently we are able to "see" these mechanisms...

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Patient Education  2 results see all →