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About 1,445 results

ALLMedicine™ Paroxysmal Nocturnal Hemoglobinuria Center

Research & Reviews  466 results

Myelodysplastic Syndromes Treatment Protocols
https://emedicine.medscape.com/article/2006494-overview

Jun 23rd, 2022 - Treatment Protocols Treatment recommendations for myelodysplastic syndromes (MDS) are based on a patient’s Revised International Prognostic Scoring System score in addition to evaluation of a patient’s performance status and molecular genetic test...

Myelodysplastic Syndromes Treatment Protocols
http://emedicine.medscape.com/article/2006494-overview

Jun 23rd, 2022 - Treatment Protocols Treatment recommendations for myelodysplastic syndromes (MDS) are based on a patient’s Revised International Prognostic Scoring System score in addition to evaluation of a patient’s performance status and molecular genetic test...

Myelodysplastic Syndromes Treatment Protocols
https://emedicine.medscape.com/article/2006494-print

Jun 23rd, 2022 - Treatment recommendations for myelodysplastic syndromes (MDS) are based on a patient’s Revised International Prognostic Scoring System score in addition to evaluation of a patient’s performance status and molecular genetic testing (eg, for 5q31 de...

Pharmacokinetics and pharmacodynamics of pozelimab alone or in combination with cemdisi...
https://doi.org/10.1371/journal.pone.0269749
PloS One; Devalaraja-Narashimha K, Huang C et. al.

Jun 17th, 2022 - Paroxysmal nocturnal hemoglobinuria (PNH) is a rare disease caused by uncontrolled complement activation; effective and approved treatments include terminal complement inhibition. This study assessed whether combination cemdisiran (an investigatio...

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

Jun 16th, 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...

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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  30 results

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

Jun 16th, 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 Study Evaluating The Efficacy And Safety Of Crovalimab Versus Eculizumab In Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Complement Inhibitors.
https://clinicaltrials.gov/ct2/show/NCT04432584

Jun 9th, 2022 - A study designed to evaluate the non-inferiority of crovalimab compared with eculizumab in participants with PNH currently treated with complement inhibitors. This study will enroll approximately 250 participants.

Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria
https://clinicaltrials.gov/ct2/show/NCT04747613

Jun 9th, 2022 - The purpose of this phase 3 open-label, single arm, multicenter study is to evaluate the long-term safety, tolerability and efficacy of iptacopan in patients with PNH and to provide access to patients who have completed (without tapering down) Pha...

A Study of Ravulizumab (ALXN1210) in Children and Adolescents With Paroxysmal Nocturnal Hemoglobinuria
https://clinicaltrials.gov/ct2/show/NCT03406507

May 9th, 2022 - The study consists of a 4-week Screening Period, a 26-week Primary Evaluation Period, and an Extension Period of up to 4 years (with the exception of any country-specific mandates), whichever occurs first. Efficacy and safety data are reported for...

Ravulizumab-Controlled Study to Evaluate the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria Who Are Complement Inhibi...
https://clinicaltrials.gov/ct2/show/NCT05133531

May 9th, 2022 - The primary objective of the study is: To evaluate the effect on hemolysis and red blood cells (RBC) transfusions over a 24-week treatment period of pozelimab and cemdisiran combination treatment versus ravulizumab treatment in patients with activ...

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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 →