Tejas Jain*

Coventry University, England

*Corresponding Author: Tejas Jain, Coventry University, England.

Received: June 26, 2025; Published: July 30, 2025

Abstract

Another name for Fabry disease (FD) is Anderson-Fabry disease. Changes in the GLA gene cause globotriaosylceramide (Gb3) to accumulate and α-galactosidase A (α-Gal A) to be deficient in FD, an orphan X-linked lysosomal storage disorder (LSD). Consequently, multiple organs fail, particularly the heart, kidneys and neurological system. Present therapies include pharmaceutical chaperone therapy (PCT) and enzyme replacement therapy (ERT). An example of PCT is migalastat (Galafold), an oral chaperone that stabilizes certain α-Gal A mutations. The goal of emerging therapeutics like gene therapy and drug delivery based on nanotechnology is to enhance treatment results. The pathophysiology, clinical presentations, and therapeutic developments of FD are examined in this review.

Keywords: Lysosomal Storage; Galafold; Alpha-Gal A; Fabry Disease; Enzyme Replacement Therapy; Nanotechnology

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Citation

Citation: Tejas Jain. “Precision Therapy in Fabry Disease: Evaluating Migalastat’s Impact on Cardiac, Renal, and Neurological Outcomes"Acta Scientific Ophthalmology 8.8 (2025): 10-15.

Copyright

Copyright: © 2025 Tejas Jain. This is an open-access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited.