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VEXAS (Vacuoles, E1 enzyme, X-linked, Autoinflammatory, Somatic) syndrome is a severe systemic inflammatory disorder caused by somatic UBA1 mutations in hematopoietic cells [1]. Current treatments, including steroids, inhibitors of IL1, IL6, and JAK family kinases, hypomethylating agents, and allogeneic stem cell transplantation, offer only partial and inconsistent disease control, with significant toxicity and frequent treatment failure [2,3,4,5,6].
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