P0068APABETALONE, A CLINICAL STAGE BET INHIBITOR, REDUCES THE PRO-INFLAMMATORY RESPONSE IN PBMCS FROM FABRY DISEASE PATIENTS ON ERT THERAPY
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DOI: 10.1093/ndt/gfaa142.p0068
Abstract: Fabry disease (FD) is a rare X-linked recessive genetic disorder caused by mutations in the α-galactosidase A gene. Absence or defects of this lysosomal enzyme lead to globotriaosylceramide (Gb3) accumulation. Gb3 disrupts basic cellular metabolic… read more here.
Keywords: pbmcs; ert therapy; patients ert; gene ... See more keywords