Patent No. EP4176877 (titled "Methods Of Enhancing And/Or Stabilizing Cardiac Function In Patients With Fabry Disease") was filed by Amicus Therapeutics on Aug 28, 2018. The application was issued on Dec 4, 2024.
Treatment of Fabry disease in patients who have previously received enzyme replacement therapy (ERT) using migalastat, a pharmacological chaperone that selectively stabilizes mutant forms of the lysosomal enzyme alpha-galactosidase A (α-Gal A). The treatment enhances cardiac function in patients with left ventricular hypertrophy, particularly in those with midwall fractional shortening (MWFS) values indicative of hypertensive target-organ damage. Migalastat is administered orally at a dose of 123 mg every other day, and its efficacy is demonstrated in patients with a range of Fabry disease severity, including those with primarily cardiac manifestations.

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