Amicus Therapeutics Inc.

Clinical research in Fabry disease centers on patients with amenable GLA variants, including adults and children, with attention to treatment use in the setting of renal impairment and end-stage renal disease requiring hemodialysis. The sponsor also studies migalastat in populations where kidney function may affect therapeutic management.

• Pharmacokinetics of migalastat
• Severe renal impairment
• End-stage renal disease
• Pediatric Fabry disease

Interest extends across age groups, with pediatric evaluation of Fabry disease and ongoing attention to treatment tolerability in metabolically and renally complex patients.