Small-molecule factor B inhibitor for the treatment of complement-mediated diseases

Schubart, Anna; Anderson, Karen; Mainolfi, Nello; Sellner, Holger; Ehara, Takeru; M. Adams, Christopher; Mac Sweeney, Aengus; Liao, Sha-Mei; Crowley, Maura; Littlewood-Evans, Amanda; Sarret, Sophie; Wieczorek, Grazyna; Perrot, Ludovic; Dubost, Valérie; Flandre, Thierry; Zhang, Yuzhou; J. H. Smith, Richard; M. Risitano, Antonio; G. Karki, Rajeshri; Zhang, Chun; Valeur, Eric; Sirockin, Finton; Gerhartz, Bernd; Erbel, Paulus; Hughes, Nicola; M. Smith, Thomas; Cumin, Frederic; A. Argikar, Upendra; Haraldsson, Börje; Mogi, Muneto; Sedrani, Richard; Wiesmann, Christian; Jaffee, Bruce; Maibaum, Jürgen; Flohr, Stefanie; Harrison, Richard; Eder, Jörg

doi:10.1073/pnas.1820892116

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Small-molecule factor B inhibitor for the treatment of complement-mediated diseases

Shared by NobleBlocks on Mar 28, 2019 • 12:00 AM UTC

Authors:

Anna Schubart

Karen Anderson

Nello Mainolfi

Abstract

Dysregulation of the alternative complement pathway (AP) predisposes individuals to a number of diseases including paroxysmal nocturnal hemoglobinuria, atypical hemolytic uremic syndrome, and C3 glomerulopathy. Moreover, glomerular Ig deposits can lead to complement-driven nephropathies. Here we des...

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