Patent No. EP3914247 (titled "Use Of Migalostat In Reducing The Risk Of Cerebrovascular Event In Patients With Fabry Disease") was filed by Amicus Therapeutics on Jan 22, 2020. The application was issued on Nov 13, 2024.
Treatment of Fabry disease using migalastat, a pharmacological chaperone that stabilizes mutant forms of the lysosomal enzyme alpha-galactosidase A (α-Gal A). Migalastat selectively binds to α-Gal A and facilitates its proper trafficking to lysosomes, reducing the accumulation of globotriaosylceramide (GL-3) and related glycosphingolipids. The treatment is effective in both ERT-naive and ERT-experienced patients, and has been shown to reduce the risk of cerebrovascular events.

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