Fabry disease biomarkers in patientsswitched from enzyme replacement therapyto migalastat oral chaperone therapy: supplementary table 1

Background: A biomarker profile was evaluated longitudinally in patients with Fabry disease switchedfrom enzyme replacement therapy (ERT) to migalastat. Methods: Sixteen Gb3 isoforms and eight lyso-Gb3 analogues were analyzed in plasma and urine by LC–MS/MS at baseline and at three different timepoints in naive participants and participants switching from either agalsidase α or β to migalastat. Results:Twenty-nine adult participants were recruited internationally (seven centers). The Mainz Severity ScoreIndex and mean biomarker levels remained stable (p ≥ 0.05) over a minimum of 12 months comparedwith baseline following the treatment switch. Conclusion: In this cohort of patientswith Fabry diseasewithamenable mutations, in the short term, a switch from ERT to migalastat did not have a marked effect onthe average biomarker profile.