ALLMedicine™ Paroxysmal Nocturnal Hemoglobinuria Center
Research & Reviews 523 results
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, ...
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)...
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...
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...
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...
Guidelines 1 results
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....
Drugs 6 results see all →
Clinicaltrials.gov 84 results
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, ...
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)...
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...
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...
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...
News 73 results
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...
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 ...
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...
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).
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...
