Background Fabry disease, an inherited lysosomal storage disorder, causes multi-organ
pathology resulting in substantial morbidity and a reduced life expectancy. Although Fabry …

Fabry disease is an inherited X-linked disorder that presents during childhood in male and
female patients. Young patients may initially experience pain, hypohidrosis, and …

Patients with Fabry disease (FD) are at a high risk for developing chronic kidney disease
and cardiovascular disease. The availability of specific but costly therapy has elevated the …

Fabry disease (FD), a rare X‐linked disease, can be treated with bi‐monthly infusion of
enzyme replacement therapy (ERT) to replace deficient α‐galactosidase A (AGAL‐A). ERT …

Fabry disease is an X-linked lysosomal storage disorder caused by the accumulation of
glycosphingolipids in various tissues and body fluids, leading to progressive organ damage …

Nephrotoxicity is a critical adverse event that leads to discontinuation of kinase inhibitor (KI)
treatment. Here we show, through meta-analyses of FDA Adverse Event Reporting System …

Results No statistical differences were found in baseline or final GFR or albuminuria. Kidney
biopsies showed significant reduction of podocyte globotriaosylceramide in both the lower …

Fabry disease (FD) is a rare, X-linked disorder caused by mutations in the GLA gene
encoding the enzyme α-galactosidase A. Complete or partial deficiency in this enzyme leads …

Background Fabry disease is caused by a deficiency of the lysosomal enzyme α-
galactosidase, resulting in progressive accumulation of globotriaosylceramide (GL-3). The …

Fabry disease is a rare X-linked lysosomal storage disorder due to mutations in the GLA
gene causing complete or partial deficiency of the enzyme a-galactosidase A (a-Gal A), and …