Clinical Research Directory

Inclusion Criteria: * Confirmed GLA mutation predicted to be responsive migalastat in the human embryonic kidney (HEK-293) cell-based assay * At least 2 years of age * Strong clinical indication for treatment of Fabry disease * No other treatment option including either unsuitable for ERT or unable to access ERT * Appropriate female and male contraception * Willing to receive treatment with migalastat HCl via this program including having signed an authorization for sharing clinical data Exclusion Criteria: * Scheduled for renal or other organ transplant or replacement therapy * Receiving GLYSET® (miglitol), ZAVESCA® (miglustat) or enzyme replacement therapy FABRAZYME® (agalsidase beta), REPLAGAL™ (agalsidase alpha), or Elfabrio® (pegunigalsidase alfa) * Contraindication to migalastat, i.e., sensitivity to other iminosugar such as miglustat, miglitol * Treated with another investigational drug within 30 days of start of migalastat HCl treatment * Unable to comply with study requirements or deemed otherwise unsuitable for study entry in the opinion of the investigator.