Effects of Current Therapies on Disease Progression in Fabry Disease: A Narrative Review for Better Patient Management in Clinical Practice
- Published on 03/07/2025
- Reading time: 12 min.
Renzo Mignani 1 , Elena Biagini 2 ,3 , Maurizio Pieroni 9
1 Nephrology, Dialysis and Transplantation, IRCCS S. Orsola Hospital Bologna, University of Bologna, Bologna, Italy
2 Cardiology Unit, Cardiac Thoracic and Vascular Department, IRCCS Azienda Ospedaliero-Universitaria di Bologna, Bologna, Italy
3 European Reference Network for Rare, Low Prevalence and Complex Diseases of the Heart-ERN GUARD-Heart), Bologna, Italy
9 Cardiovascular Department, ASL8 Arezzo San Donato Hospital, Via Pietro Nenni 20, 52100, Arezzo, Italy
Abstract
Fabry disease (FD) is a rare lysosomal storage disorder that is characterized by renal, neurological, and cardiovascular dysfunction. Four treatments are currently available for patients with FD; three enzyme replacement therapies (ERTs; agalsidase alfa, agalsidase beta, and pegunigalsidase alfa) and one pharmacological chaperone (migalastat). This review focuses on the evidence for the benefits of ERTs and migalastat, and provides an overview of their impact on disease manifestations...
To continue reading this article in Full-Text...
Peer-Reviewed Journals A-Z
Search | Advanced search
Get the latest news in Rare Diseases
Receive our newsletter to stay up to date with the latest news in Rare Diseases
