Methods of treating Fabry patients having renal impairment
AssigneeAmicus Therapeutics, Inc.
InventorsJeff Castelli, Elfrida Benjamin
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Abstract
Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in α-galactosidase A. Certain methods comprise administering migalastat or a salt thereof every other day, such as administering about 150 mg of migalastat hydrochloride every other day.
Claims30
A molecule comprising migalastat bound to an α-galactosidase A protein comprising a HEK assay amenable mutation selected from the group consisting of D33G, L36W, Q57L, M96I, R112G, C174R, M187I, I253S, G258R, G271S/D313Y, D313Y, D322E, G325R, and R356Q.
An α-galactosidase A protein having a HEK amenable mutation selected from the group consisting of D33G, L36W, Q57L, M96I, R112G, C174R, M187I, I253S, G258R, G271S/D313Y, D313Y, D322E, G325R, and R356Q,
- wherein the protein has increased stability as compared to a naturally-occurring α-galactosidase A protein having the same mutation.
Dependencies are inferred from the claim text.