In patients with Fabry disease, a pathogenic variant in the galactosidase alpha (GLA) gene leads to α-galactosidase A (aGAL-A) enzyme deficiency, which in turn results in a progressive ...
CloseCurlyDoubleQuote; In patients with Fabry disease, a pathogenic variant in the galactosidase alpha (GLA) gene leads to a-galactosidase A (aGAL-A) enzyme deficiency, which in turn results in a ...
It remains to be seen whether other enzyme-replacement therapies can duplicate its economic success. Fabry's disease is an X-linked disorder caused by a deficiency of α-galactosidase. The ...