Results 1 to 10 of about 18,676 (150)

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   +4 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

Efficacy and safety of the investigational complement C5 inhibitor zilucoplan in patients hospitalized with Covid-19: an open-label randomized controlled trial [PDF]

Respir Res, 2022
Abstract Background: The efficacy and safety of complement inhibition in Covid-19 patients is unclear.Methods: A multicenter randomized controlled, open-label trial. Hospitalized Covid-19 patients with signs of hyperinflammation and hypoxemia (PaO2/FiO2 below 350 mHg) were randomized (2:1 ratio) to receive standard of care with or without the ...
De Leeuw E, Damme KFV, Declercq J, Bosteels C, Maes B, Tavernier SJ, Detalle L, Smart T, Glatt S, Debeuf N, Deckers J, Lameire S, Vandecasteele SJ, De Neve N, Demedts IK, Govaerts E, Knoop C, Vanhove K, Moutschen M, Terryn W, Depuydt P, Braeckel EV, Haerynck F, Hendrickx TC, Parrein V, Lalla M, Brittain C, Lambrecht BN.   +27 more
europepmc   +8 more sources

One-year efficacy and safety of ravulizumab in adults with paroxysmal nocturnal hemoglobinuria naïve to complement inhibitor therapy: open-label extension of a randomized study

Therapeutic Advances in Hematology, 2020
Background: Ravulizumab, the only long-acting complement C5 inhibitor for adults with paroxysmal nocturnal hemoglobinuria (PNH), demonstrated non-inferiority to eculizumab after 26 weeks of treatment in complement inhibitor-naïve patients during a phase ...
Hubert Schrezenmeier, Austin G Kulasekararaj, Lindsay Mitchell   +2 more
exaly   +2 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

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

Therapeutic advances in eculizumab for atypical hemolytic uremic syndrome: a narrative review [PDF]

Frontiers in Pharmacology
Atypical hemolytic uremic syndrome (aHUS) represents an uncommon, life-threatening complement-mediated thrombotic microangiopathy that is linked to both significant renal and extrarenal complications.
Dandan Zhang, Yichen Xiao, Hualin Ma, Hualin Ma, Hualin Ma   +4 more
doaj   +2 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

Optimizing Care in Patients with Paroxysmal Nocturnal Hemoglobinuria: Managing Suboptimal Response and Uncontrolled Disease [PDF]

Journal of Blood Medicine
Marc Bienz,1 Monika Oliver,2 Catherine Sperlich,3 Christopher J Patriquin4 1Division of Hematology, Jewish General Hospital, Montreal, QC, Canada; 2Division of Hematology, University of Alberta Hospital, Edmonton, AB, Canada; 3Division of hematology and ...
Bienz M, Oliver M, Sperlich C, Patriquin CJ   +3 more
doaj   +2 more sources