U.S., Aug. 6 -- ClinicalTrials.gov registry received information related to the study (NCT07746986) titled 'DECIPHERING AND TARGETING VEXAS' on July 08.
Brief Summary: VEXAS (vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic) syndrome (1/4,000 males aged >50) is a paradigmatic age-onset acquired, severe, autoinflammatory hematological disease due to clonal dominance of hematopoietic cells bearing a somatic mutation in the UBA1 (ubiquitin-activating enzyme 1) gene. VEXAS presents with treatment-refractory systemic myeloid-driven inflammatory manifestations and hematologic abnormalities, hence the poor prognosis. Current available therapies are poorly effective and burdened by debilitating side effects. VEXAS mutations inactivate cyto...