agalsidase beta

Fabry disease, an X-linked inherited lysosomal storage disorder, is caused by mutations in the gene encoding α-galactosidase, GLA… Expand

Background:In 2009, the agalsidase beta shortage resulted in switching to agalsidase alfa treatment for many Fabry disease… Expand

BACKGROUND Fabry disease, an X-linked lysosomal storage disorder, causes intracellular accumulation of glycosphingolipids leading… Expand

Background. The purpose of this study was to identify determinants of renal disease progression in adults with Fabry disease… Expand

The enzyme replacement therapy agalsidase alfa (Replagal®) has an amino acid sequence identical to that of native α-galactosidase… Expand

The enzyme replacement therapy agalsidase alfa (Replagal®) has an amino acid sequence identical to that of native α-galactosidase… Expand

PurposeAnderson-Fabry disease is a multisystemic disorder of lipid metabolism secondary to X-chromosome alterations and is… Expand

Disclosure: The Fabry Registry is sponsored by Genzyme Corporation. U.F.-R., K.S., R.J.H., M.B., A.M., and A.P.B. serve on the… Expand

Context Fabry disease is an X-linked storage disorder characterized by deficient lysosomal enzyme activity and excessive… Expand

Gastrointestinal symptoms are often an early and prominent manifestation of Fabry disease, an X-linked inborn error of metabolism… Expand