A systematic review on safety and efficacy of Migalastat for the treatment of Fabry's disease.
Haya MajidNeharika VermaShivani BhandariSparsh GuptaNidhi Bharal AgarwalPublished in: Expert opinion on pharmacotherapy (2024)
Migalastat showed varied effects on enzyme activity and substrate levels, with gender-specific differences noted in GL-3 substrate activity and eGFR. Overall, it improved cardiac and renal outcomes similarly to enzyme replacement therapy, with a comparable safety profile.