Results 1 to 10 of about 1,060,485 (284)

The complement C5 inhibitor crovalimab in paroxysmal nocturnal hemoglobinuria. [PDF]

Blood, 2020
Abstract Complement C5 inhibition is the standard of care (SoC) for patients with paroxysmal nocturnal hemoglobinuria (PNH) with significant clinical symptoms. Constant and complete suppression of the terminal complement pathway and the high serum concentration of C5 pose challenges to drug development that result in IV-only treatment ...
Röth A, Nishimura JI, Nagy Z, Gaàl-Weisinger J, Panse J, Yoon SS, Egyed M, Ichikawa S, Ito Y, Kim JS, Ninomiya H, Schrezenmeier H, Sica S, Usuki K, Sicre de Fontbrune F, Soret J, Sostelly A, Higginson J, Dieckmann A, Gentile B, Anzures-Cabrera J, Shinomiya K, Jordan G, Biedzka-Sarek M, Klughammer B, Jahreis A, Bucher C, Peffault de Latour R.   +27 more
europepmc   +6 more sources

Complement C5 Inhibitor Ameliorates a Case of Dysferlinopathy. [PDF]

Neurol Neuroimmunol Neuroinflamm
Dysferlinopathy is an autosomal recessive muscular dystrophy caused by pathogenic variants of the DYSF gene. Currently, no clinical effective treatment is available. Given the myopathologic and animal model evidence on complement activation in dysferlinopathy, we explored the potential therapeutic effect of complement inhibition.We reported a case of ...
Kang S, Wang Q, Lv H, Deng J, Zhang W, Lu X, Yuan Y, Wang Z, Zheng F, Yu M.   +9 more
europepmc   +3 more sources

Efficacy and Safety of Ravulizumab, a Complement C5 Inhibitor, in Adults With Amyotrophic Lateral Sclerosis: A Randomized Clinical Trial.

JAMA Neurol, 2023
ImportanceAdditional therapies for amyotrophic lateral sclerosis (ALS) are urgently needed. Immune-mediated complement activation may be involved in ALS pathogenesis as evidenced by the upregulation of terminal components; thus, complement inhibition could potentially slow progression.ObjectiveTo evaluate the safety and efficacy of the terminal ...
Genge A, van den Berg LH, Frick G, Han S, Abikoff C, Simmons A, Lin Q, Patra K, Kupperman E, Berry JD.   +9 more
europepmc   +5 more sources

Complement-targeted therapies for transplant-associated thrombotic microangiopathy: recent advances [PDF]

Frontiers in Pharmacology
Transplant-associated thrombotic microangiopathy (TA-TMA) is a life-threatening complication of hematopoietic stem cell transplantation in which systemic complement activation induces endothelial injury, leading to microangiopathic hemolytic anemia ...
Taichiro Tokura, Taichiro Tokura, Sayuri Motomura   +2 more
doaj   +2 more sources

In vivo evidence of effective complement modulation by TriFu in comparison to C5 inhibition and complement depletion [PDF]

Blood Advances
: The therapeutic landscape of complement inhibition has recently expanded considerably. However, most approved agents specifically target single complement components systemically, which can result in limited functional effects.
Susa Savukoski, Alina Maria Dettner, Anke Schultze, Christoph Gerber, Michael Stefan Steinberger, Rebecca Halbgebauer, Peter Melter, Arthur Dopler, Annette Palmer, Christian Karl Braun, Susanne Hafner, Bo Nilsson, Kristina Nilsson-Ekdahl, Tom Eirik Mollnes, Raban Heller, Hubert Schrezenmeier, Christoph Q. Schmidt, Markus S. Huber-Lang, Marco Mannes   +18 more
doaj   +2 more sources

Use of complement C5-inhibitor eculizumab in patients with infection-associated hemolytic uremic syndrome - a case-series report. [PDF]

BMC Pediatr
Abstract Background: Hemolytic uremic syndrome (HUS), characterized by the triad of microangiopathic hemolytic anemia, thrombocytopenia and acute kidney injury (AKI), remains a leading cause of pediatric AKI. The complement system has a crucial role in the pathogenesis of atypical hemolytic uremic syndrome (aHUS) and eculizumab (ECZ) was ...
Varga P, Biró E, Berkes A, Lakatos E, Szikszay E, Prohászka Z, Szabó T.   +6 more
europepmc   +4 more sources

Zilucoplan, a macrocyclic peptide inhibitor of human complement component 5, uses a dual mode of action to prevent terminal complement pathway activation

Frontiers in Immunology, 2023
IntroductionThe complement system is a key component of the innate immune system, and its aberrant activation underlies the pathophysiology of various diseases.
Guo-Qing Tang, Yalan Tang, Ketki Dhamnaskar, Michelle D. Hoarty, Rohit Vyasamneni, Douangsone D. Vadysirisack, Zhong Ma, Nanqun Zhu, Jian-Guo Wang, Charlie Bu, Bestine Cong, Elizabeth Palmer, Petra W. Duda, Camil Sayegh, Alonso Ricardo   +14 more
doaj   +3 more sources

Complement C5 inhibition with eculizumab or ravulizumab is associated with increased cardiovascular and thromboembolic risk after ST-elevation myocardial infarction: a propensity-matched global retrospective cohort study [PDF]

BMC Cardiovascular Disorders
Background Complement-mediated myocardial and vascular injury is a hallmark of ST-elevation myocardial infarction (STEMI). Dysregulated activation of complement component C5 has been implicated in endothelial dysfunction, exacerbated inflammatory ...
Carl Vahldieck
doaj   +2 more sources

Effectiveness of Iptacopan Versus C5 Inhibitors in Complement Inhibitor‐Naive Patients With Paroxysmal Nocturnal Haemoglobinuria

eJHaem
Background Paroxysmal nocturnal haemoglobinuria (PNH) is characterised by haemolytic anaemia, bone marrow failure and thrombosis. The single‐arm phase 3 APPOINT‐PNH trial (NCT04820530) investigating iptacopan monotherapy in complement inhibitor‐naive ...
Matthew Holt, Richard J. Kelly, Jilles M. Fermont, Soudeh Ansari, Marion Dahlke, Isabelle Brindel, Lynda Maafa, Flore Sicre deFontbrune, Régis Peffault deLatour   +8 more
doaj   +3 more sources

Novel interactions between the C5‐C5aR1 Axis and IF1: Implications for kidney mitochondrial physiology and ischemia–reperfusion injury [PDF]

Physiological Reports
Ischemia‐reperfusion injury (IRI) is a prevalent condition that predominantly afflicts hospitalized patients, inducing acute kidney injury (AKI). In recent years, complement 5 (C5) and its anaphylatoxin receptor C5aR1 have been implicated in driving ...
Madison McGraw, Amod Sharma, Dinesh Bhattarai, Neriman Gokden, LeeAnn Macmillan‐Crow, Nirmala Parajuli   +5 more
doaj   +2 more sources