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 ...

C3G and IC-MPGN can be difficult to diagnose because they are so rare and their symptoms are vague and nonspecific. A kidney biopsy is considered the gold standard and usually needed to confirm the ...

aRheumatology Service, Maimonides Institute of Biomedical Research of Cordoba (IMIBIC), Reina Sofia University Hospital, University of Córdoba, Córdoba, Spain Antiphospholipid syndrome is a rare ...

Among people with sickle cell disease—an inherited blood disorder—acute chest syndrome (ACS) is the leading cause of death. The condition is the result of red blood cells becoming stiff, sticky, and ...

Sickle cell disease can lead to a severe complication known as acute chest syndrome (ACS), but the underlying mechanisms are not well understood. A new study by Mass General Brigham investigators ...

ARO-C3 treatment led to deep and sustained reductions in alternative pathway complement activity and proteinuria. ARO-C3, an investigational RNA interference-based therapy targeting hepatic expression ...

TSP-1 reduced C3 and C5 cleavage and formation of the membrane attack complex, attenuating complement-mediated injury in AAV models. Complement activation is a relevant driver in the pathomechanisms ...

Building a neuromuscular franchise with DNTH103 in gMG, CIDP and MMN with a potential best-in-class, potent classical pathway inhibitor intended as a self-administered autoinjector dosed once every ...