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US 2020/0206204 A1Published application
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.
Claims23
A method of stabilizing renal function in a patient diagnosed with Fabry disease and having renal impairment, the method comprising:
- a. administering to the patient an effective amount of migalastat or salt thereof at a frequency of once every other day, and
- b. reducing left ventricular mass index (LVMi) in the patient,
- wherein the effective amount is about 100 mg to about 150 mg free base equivalent (FBE), wherein the administration of the migalastat or salt thereof is effective to stabilize renal function in the patient and wherein the patient has a HEK assay amenable mutation in α-galactosidase A.
Dependencies are inferred from the claim text.