Efficacy and safety of targeted therapies in VEXAS syndrome: retrospective study from the FRENVEX.
Annals of the rheumatic diseasesMay 2024
Description
Vacuoles, E1 enzyme, X-linked, autoinflammatory and somatic (VEXAS) syndrome is an adult-onset autoinflammatory disease associated with somatic ubiquitin-like modifier-activating enzyme 1 (UBA1) mutations. We aimed to evaluate the efficacy and safety of targeted therapies.