Left untreated, PNH could cause death in about half of patients within 10 years of diagnosis. The primary cause of PNH-related mortality is thromboembolic events. A host of anti-complement strategies, ...

The treatment landscape of generalised myasthenia gravis, a prototypic antibody-mediated disorder of the neuromuscular junction, has considerably evolved in the past 8 years.1 The approval of targeted ...

The Food and Drug Administration (FDA) has accepted for Priority Review the supplemental Biologics License Application (sBLA) for Soliris (eculizumab; Alexion) for use in patients with neuromyelitis ...

Arrowhead Pharmaceuticals (NASDAQ:ARWR) reported interim data from Part 1 of an ongoing phase 1/2 trial of ARO-C3 in healthy people as a potential therapy for complement mediated diseases. The ...

Complement C5a is a known mediator of inflammatory and neuropathic pain. In a new study, patients with NMOSD who were in remission and healthy controls were asked to rate their pain on a visual analog ...

A rare blood disorder treatable with infused medications now has its first oral drug alternative. FDA approval of the new drug from Novartis positions the pill to compete directly against two ...

Novartis AG has identified spirocyclic piperidinyl derivatives acting as complement factor B (CFB) inhibitors reported to be useful for the treatment of age-related macular degeneration (AMD), ...

As an academic pediatrician-scientist, my overarching career goals are to advance knowledge that will improve the health of children with bleeding and clotting disorders and educate the next ...