Autoinflammatory diseases were first recognized nearly 20 years ago as distinct clinical and
immunological entities caused by dysregulation in the innate immune system. Since then …

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare bone marrow failure disorder that
manifests with hemolytic anemia, thrombosis, and peripheral blood cytopenias. The …

Background Persistent hemolytic anemia and a lack of oral treatments are challenges for
patients with paroxysmal nocturnal hemoglobinuria who have received anti-C5 therapy or …

Background Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired disease
characterized by chronic complement-mediated hemolysis. C5 inhibition controls …

Congenital disorders of glycosylation are a genetically and clinically heterogeneous group
of> 130 diseases caused by defects in various steps along glycan modification pathways …

The treatment of paroxysmal nocturnal hemoglobinuria has been revolutionized by the
introduction of the anti-C5 agent eculizumab; however, eculizumab is not the cure for …

Glycosylation produces an abundant, diverse, and highly regulated repertoire of cellular
glycans that are frequently attached to proteins and lipids. The past decade of research on …

Intravascular haemolysis is a fundamental feature of chronic hereditary and acquired
haemolytic anaemias, including those associated with haemoglobinopathies, complement …

The complement system is central to first-line defense against invading pathogens.
However, excessive complement activation and/or the loss of complement regulation …

Background In the PEGASUS trial, the complement C3 inhibitor, pegcetacoplan, showed
superiority to eculizumab in improving haematological outcomes in adult patients with …