A Systematic Review on Safety and Efficacy of Migalastat for the treatment of Fabry's Disease.

Haya MajidNeharika VermaShivani BhandariSparsh GuptaNidhi Bharal Agarwal

Published 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.

Keyphrases

replacement therapy
smoking cessation
small cell lung cancer
epidermal growth factor receptor
tyrosine kinase
mental health
heart failure
amino acid
structural basis
adipose tissue