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Migalastat Approved DB05018
small molecule | CAS: 108147-54-2

Fabry disease is a rare, progressive genetic disorder characterized by a defective GLA gene that causes a deficiency in the enzyme alpha-Galactosidase A (alpha-Gal A). This enzyme is responsible for breaking down glycosphingolipid substrate that, when deficie…

Kategori:
Alimentary Tract and MetabolismAlkaloidsAlpha-Galactosidase A (alpha-Gal A) Pharmacological Chaperones +6
Target Protein:
Alpha-galactosidase A
Waktu ParuhThe mean eliminatio…
Vol. DistribusiIn healthy voluntee…
KlirensFollowing ascending…
Genetik -