Population PK-PD Modeling of Danicopan Add-On Therapy in Participants With Paroxysmal Nocturnal Hemoglobinuria Treated With Ravulizumab or Eculizumab. [PDF]
ABSTRACT Danicopan is a first‐in‐class orally administered complement factor D inhibitor, approved as an add‐on therapy to ravulizumab or eculizumab for the treatment of clinically significant extravascular hemolysis in patients with paroxysmal nocturnal hemoglobinuria (PNH).
Chen J +4 more
europepmc +2 more sources
Successful management of unstable angina in a ravulizumab-treated patient with paroxysmal nocturnal hemoglobinuria [PDF]
Ravulizumab is an anti-C5 antibody approved for treating paroxysmal nocturnal hemoglobinuria (PNH). In August 2019, a 77-year-old Japanese man with PNH, who had been on ravulizumab treatment for 2 years, was hospitalized for chest discomfort and malaise.
Fukatsu, Masahiko +21 more
core +1 more source
Long-Term Efficacy and Safety of the Long-Acting Complement C5 Inhibitor Ravulizumab for the Treatment of Atypical Hemolytic Uremic Syndrome in Adults [PDF]
Hemolytic uremic syndrome; Kidney failure; RavulizumabSíndrome hemolítico urémico; Insuficiencia renal; RavulizumabSíndrome hemolític urèmic; Insuficiència renal; RavulizumabIntroduction Atypical hemolytic uremic syndrome (aHUS) is a rare, complex ...
Ariceta Iraola, Gema +5 more
core +1 more source
The potential of individualized dosing of ravulizumab to improve patient-friendliness of paroxysmal nocturnal haemoglobinuria treatment at reduced costs [PDF]
Contains fulltext : 237767.pdf (Publisher’s version ) (Open Access)Ravulizumab is a very expensive complement C5-inhibitor for the treatment of paroxysmal nocturnal haemoglobinuria, with a fixed-dosing interval of 8 weeks.
Avest, M. ter +7 more
core +1 more source
Summary of Research: Terminal Complement Inhibitor Ravulizumab in Generalized Myasthenia Gravis
This article provides a summary of a previously published paper: Terminal Complement Inhibitor Ravulizumab in Generalized Myasthenia Gravis. The paper reported the results of the CHAMPION-MG trial which investigated the drug ravulizumab in the rare ...
Tuan Vu +9 more
doaj +1 more source
Rationale and Objective: Ravulizumab and eculizumab have shown efficacy for the treatment of atypical hemolytic uremic syndrome (aHUS), but real-world evidence for ravulizumab is limited owing to its more recent approval.
Yan Wang +5 more
doaj +1 more source
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 +11 more
doaj +1 more source
Design and Rationale of the APPELHUS Phase 3 Open-Label Study of Factor B Inhibitor Iptacopan for Atypical Hemolytic Uremic Syndrome. [PDF]
Atypical hemolytic uremic syndrome (aHUS) is a rare, progressive, and life-threatening form of thrombotic microangiopathy (TMA) which is caused by dysregulation of the alternative complement pathway (AP). Complement inhibition is an effective therapeutic
Bagga, A. +8 more
core +1 more source
Complement Mediated Hemolytic Anemias in the COVID-19 Era : Case Series and Review of the Literature [PDF]
The complex pathophysiologic interplay between SARS-CoV-2 infection and complement activation is the subject of active investigation. It is clinically mirrored by the occurrence of exacerbations of complement mediated diseases during COVID-19 infection ...
Barcellini W. +4 more
core +1 more source
CADTH reimbursement reviews are comprehensive assessments of the clinical effectiveness and cost-effectiveness, as well as patient and clinician perspectives, of a drug or drug class. The assessments inform non-binding recommendations that help guide the reimbursement decisions of Canada's federal, provincial, and territorial governments ...
openaire +2 more sources

