Figure 3.
Work-Up to Assess Clinical Amenability in Patients Treated with Migalastat
Biochemical response after migalastat initiation should already be measurable after 3 months of treatment by increasing AGAL activities and decreasing plasma lyso-Gb3 levels. Regular follow-up examinations should include echocardiography of the heart to assess left ventricular hypertrophy (LVH) and left ventricular masses (LVMs), determination of eGFR and proteinuria/albuminuria measures for kidney function, brain MRI to assess cerebrovascular events (CVEs) and white matter lesions (WMLs), a pain questionnaire for FD-specific pain and gastrointestinal symptoms, and α-galactosidase A (AGAL) activity and globotriaosylsphingosine (lyso-Gb3) measurements in blood.