Results 31 to 40 of about 898 (115)

Iptacopan in Idiopathic Immune Complex–Mediated Membranoproliferative Glomerulonephritis: Protocol of the APPARENT Multicenter, Randomized Phase 3 Study [PDF]

, 2023
\ua9 2023Introduction: Immune complex–mediated membranoproliferative glomerulonephritis (IC-MPGN) is an ultra-rare, fast-progressing kidney disease that may be idiopathic (primary) or secondary to chronic infection, autoimmune disorders, or monoclonal ...
Vivarelli M, Bomback AS, Webb NJA, Veldandi UK, Smith RJH, Meier M, Wang Y, Kavanagh D   +7 more
core   +4 more sources

Switching patients with PNH from pegcetacoplan to iptacopan: a case series

Hematology
Objectives Limited published data exist on switching therapy from pegcetacoplan to iptacopan in patients with paroxysmal nocturnal hemoglobinuria (PNH).Methods Three patient cases were collected from the Mayo Clinic, Cleveland Clinic, and Levine Cancer ...
Mark Orland, Carmelo Gurnari, Srinivasa Sanikommu, Jaroslaw Maciejewski, David Dingli   +4 more
doaj   +1 more source

First Successful Treatment of a Patient with a Primary Immune Complex-Membranoproliferative Glomerulonephritis with Iptacopan: A Case Report

Case Reports in Nephrology and Dialysis
Introduction: Nowadays, there is insufficient evidence for the recommendation of management patients with a primary membranoproliferative glomerulonephritis (MPGN).
Simone Arnold, Manuela Nickler, Michael Dickenmann, Thomas Menter, Helmut Hopfer, Patricia Hirt-Minkowski   +5 more
doaj   +1 more source

Treatment of transplantation-associated thrombotic microangiopathy with iptacopan: two cases report [PDF]

Annals of Hematology
Xiao-Dong Mo
exaly   +2 more sources

Exploring individualized crovalimab dosing in PNH through in silico modelling: Potential for improved convenience and cost efficiency

British Journal of Clinical Pharmacology, EarlyView.
Abstract Aim Paroxysmal nocturnal haemoglobinuria (PNH) is a rare, acquired haematopoietic stem cell disorder. Crovalimab, a complement C5‐inhibitor, is approved for PNH and can be self‐administered subcutaneously every 4 weeks, offering a more convenient route than intravenous C5‐inhibitors.
Mendy ter Avest, Simon E. Koele, Nicole C. A. J. van de Kar, Saskia M. C. Langemeijer, Rob ter Heine   +4 more
wiley   +1 more source

Australian consensus recommendations for the management of increased meningococcal infection risk in adults with neurological diseases treated with complement inhibitors

Internal Medicine Journal, EarlyView.
Abstract Complement inhibitor therapy carries a risk of serious infections, including meningococcal disease. Here we provide evidence‐based recommendations and expert consensus for immunisation and prophylactic treatment of patients receiving, or planning to receive, complement inhibitors for neurological conditions in the Australian setting.
Katherine A. Buzzard, Michael Barnett, Heidi Beadnall, Mirasol Forcadela, Lesley‐Ann Hall, Victoria G. Hall, Todd Hardy, Danny Hsu, Paul Kinsella, Mastura Monif, Sudarshini Ramanathan, Stephen W. Reddel, Sean Riminton, Douglas F. Johnson   +13 more
wiley   +1 more source

Breakthrough Hemolysis in Paroxysmal Nocturnal Hemoglobinuria: Mechanistic Insights and Management Strategies

Transfusion, EarlyView.
Abstract Background Paroxysmal nocturnal hemoglobinuria (PNH) is a clonal hematopoietic stem cell disorder caused by somatic mutations in the PIGA gene, resulting in loss of glycosylphosphatidylinositol (GPI)‐anchored proteins, including the complement regulatory proteins, CD55 and CD59.
Ganesh Raman, Meghana Vivek, Rida Hasan, Siobán Keel, Laura Connelly‐Smith   +4 more
wiley   +1 more source

A First‐in‐Japanese Phase 1, Double‐Blind, Placebo‐Controlled, Parallel‐Cohort Study of Sefaxersen, an Antisense Oligonucleotide Targeting Complement Factor B, in Healthy Participants

Clinical Pharmacology in Drug Development, Volume 15, Issue 9, September 2026.
Abstract Increased activity in the complement alternative pathway (AP) plays a key role in diseases such as IgA nephropathy (IgAN). This first‐in‐Japanese double‐blind Phase 1 study investigated the pharmacokinetics (PK), pharmacodynamics (PD), safety, and tolerability of sefaxersen (RO7434656), an antisense oligonucleotide targeting complement factor ...
Takakazu Sato, Hiroyuki Fukase, Takaaki Ishida, Arisa Karasawa   +3 more
wiley   +1 more source

Iptacopan monotherapy in patients with paroxysmal nocturnal hemoglobinuria: a 2-cohort open-label proof-of-concept study.

, 2022
Iptacopan (LNP023) is a novel, oral selective inhibitor of complement factor B under clinical development for paroxysmal nocturnal hemoglobinuria (PNH).
Li, Katie, Baltcheva, Irina, Jang, Jun Ho, Chawla, Raghav, Yap, Eng Soo, Ko, Bor-Sheng, Junge, Guido, Nidamarthy, Prasanna, Nidamarthy, Prasanna Kumar, Wong, Lily, Yoon, Sung-Soo   +10 more
core   +1 more source

Chiral (Stereoselective) Drugs, Asymmetric Synthesis, and Racemic Resolution Methods

Chirality, Volume 38, Issue 7, July 2026.
Chirality is crucial in drug development since both biological targets in the organism and the majority of pharmaceutical compounds are chiral. The synthesis and resolution of chiral compounds are critical steps while developing chiral drugs. Asymmetric synthesis and racemic resolution are the two most common methods for obtaining enantiopure drugs ...
Burcu Karayavuz, Cigdem Karaaslan Kirmizioglu   +1 more
wiley   +1 more source