Results 51 to 60 of about 2,146 (159)
What is this summary about? Paroxysmal nocturnal hemoglobinuria (PNH) is a rare disease where red blood cells break apart because they lack certain protective proteins.
Celso Arrais +2 more
doaj +1 more source
Across 18 randomized trials involving 1823 participants, active therapies improved both QMG and MG‐ADL outcomes versus control. Trial‐level meta‐regression showed that each 10‐year increase in mean baseline age was associated with a 1.21‐point smaller QMG benefit, explaining 25.4% of between‐trial heterogeneity; the association appeared strongest in ...
Christopher Nelke +4 more
wiley +1 more source
Low Incidence of Relapses After Vaccination in Anti‐Aquaporin‐4 Antibody‐Positive NMOSD
ABSTRACT Patients with neuromyelitis optica spectrum disorder (NMOSD) may experience increased signs and symptoms of their underlying disease when vaccinated against meningococcal disease before receiving complement component 5 inhibitor therapies. This retrospective analysis indicated an overall low relapse incidence (mean [range], 3.3% [0.7%–10.6 ...
Sean J. Pittock +4 more
wiley +1 more source
ABSTRACT Area postrema syndrome (APS) is a rare neurological syndrome that presents a significant diagnostic challenge due to its nonspecific clinical manifestations, most commonly persistent nausea and vomiting, which frequently lead to initial misdiagnosis as a gastrointestinal disorder.
Andreea‐Gabriela Manole +4 more
wiley +1 more source
IntroductionComplement component 5 inhibitor therapies (C5ITs) for rare hematological, renal, and neurological diseases are associated with increased meningococcal infection risk. Robust risk mitigation measures include vaccination, drug safety programs,
Cynthia Carrillo Infante +1 more
doaj +1 more source
Complement Factor H‐Based Therapeutics: A Comprehensive Overview
Complement factor H is a central regulator of the complement system and thus a target for therapeutic intervention. In this review, we provide an overview of past and current factor H‐based therapeutic concepts and modalities to treat complement‐mediated diseases.
Sebastiaan M. W. R. Hamers +2 more
wiley +1 more source
Abstract Paroxysmal nocturnal hemoglobinuria (PNH) is an ultra‐orphan disease. In 2026, approved complement inhibitors (CIs) for PNH include three C5 inhibitors (C5i) and three proximal inhibitors (PIs). Clinical trials for the approved PI pegcetacoplan, iptacopan, and C5i + danicopan had clear protocols for changing from terminal to PI, extrapolated ...
Morag Griffin +15 more
wiley +1 more source
Ravulizumab and Efgartigimod in Myasthenia Gravis
Finanziert durch den Open-Access-Publikationsfonds der Universität Münster.
Frauke Stascheit +16 more
openaire +4 more sources
Introduction Anti-aquaporin-4 antibody-positive (AQP4-Ab+) neuromyelitis optica spectrum disorder (NMOSD) is a complement-mediated autoimmune disease in which unpredictable and relapsing attacks on the central nervous system cause irreversible and ...
Stacey L. Clardy +10 more
doaj +1 more source

