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
left ventricular
tyrosine kinase
mental health
heart failure
structural basis
type diabetes
adipose tissue
atrial fibrillation
glycemic control