pegunigalsidase alfa (Pending FDA Approval)

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Dosing & Uses

Fabry Disease

Pending FDA approval for Fabry disease

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Pharmacology

Recombinant alpha-galactosidase A, an enzyme (deficient in Fabry disease) involved in the metabolism of glycosphingolipids (eg, GL-3); corrects abnormal glycosphingolipid metabolism resulting in reduced accumulation of glycosphingolipids and improvement of associated clinical manifestations (eg, vascular occlusion, stroke, renal failure, heart failure); reduces GL-3 deposition in capillary endothelium

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Medscape prescription drug monographs are based on FDA-approved labeling information, unless otherwise noted, combined with additional data derived from primary medical literature.