INVOLVEMENT OF ANDROGEN/ANDROGEN RECEPTOR PATHWAY IN FABRY DISEASE

摘要

Novel therapies for the treatment of Fabry disease by using androgen/androgen receptor (AR) pathway-related molecules as biomarkers and use of approaches targeting androgen/AR pathway are presented herein. The involvement of aberrant androgen/AR pathway in Fabry disease has never been previously described. The present invention describes, (i) use of approaches that target androgen/AR pathway as therapeutic treatments for Fabry disease and (2) use of the levels of androgen/AR pathway-related molecules in body fluids or tissues as biomarkers for evaluation of disease progression and efficacy of treatments in Fabry patients.