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New Fabry Disease Drug Rivals Efficacy of Enzyme Replacement

A drug developed by Amicus Therapeutics, Inc. has shown to be as effective as enzyme replacement therapy in patients with Fabry disease, according to an article published by Reuters. The drug, called migalastat HCl, recently finished a second late-stage trial, and works by binding to and aiding the alpha-Gal enzyme, which is deficient in Fabry disease patients. The deficiency causes an accumulation of a specific type of fat if left untreated, which can lead to kidney failure, heart attack, or stroke. The drug would be administered orally, which is an improvement from the current bi-weekly infusions required for enzyme replacement.