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 ...
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 ...
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 ...
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