Use Of Migalastat For Treating Fabry Disease In Pregnant Patients

Abstract

Provided are methods of treating a patient diagnosed with Fabry disease. Certain methods treat a pregnant patient with a therapeutically effective dose of migalastat or a salt thereof. Other methods treat a patient of childbearing potential with a therapeutically effective dose of migalastat or a salt thereof. Also described are the successful outcomes of pregnancies during which the pregnant patient is treated with migalastat.

1 - 30 . (canceled) 
     
     
         31 . A method for the treatment of Fabry disease in a female patient of childbearing potential, the method comprising administering to the patient a formulation comprising a therapeutically effective dose of about 100 mg to about 150 mg free base equivalent (FBE) of migalastat or a salt thereof at a frequency of once every other day, and wherein the patient is not instructed before the treatment is initiated to use effective birth control during the treatment. 
     
     
         32 . The method of  claim 31 , wherein the patient becomes pregnant after initiating the treatment. 
     
     
         33 . The method of  claim 31 , wherein the patient does not utilize effective birth control during the treatment. 
     
     
         34 . The method of  claim 32 , wherein the treatment is discontinued upon identification of the patient's pregnancy. 
     
     
         35 . The method of  claim 32 , wherein the patient's pregnancy results in a birth of a child having a gestational age of at least 37 weeks. 
     
     
         36 . The method of  claim 32 , wherein the patient's pregnancy results in a birth of a child of normal weight based on a gestational age of the child. 
     
     
         37 . The method of  claim 32 , wherein the patient's pregnancy results in a child without a birth defect. 
     
     
         38 . The method of  claim 31 , wherein the therapeutically effective dose is about 123 mg free base equivalent (FBE) of migalastat or a salt thereof. 
     
     
         39 . The method of  claim 31 , wherein the therapeutically effective dose is about 123 mg of migalastat free base. 
     
     
         40 . The method of  claim 31 , wherein the salt of migalastat is migalastat hydrochloride. 
     
     
         41 . The method of  claim 40 , wherein the therapeutically effective dose is about 150 mg of migalastat hydrochloride. 
     
     
         42 . The method of  claim 31 , wherein the formulation is administered for at least 2 weeks. 
     
     
         43 . The method of  claim 31 , wherein the formulation is administered for at least 6 weeks. 
     
     
         44 . The method of  claim 31 , wherein the formulation is administered for at least 12 weeks. 
     
     
         45 . The method of  claim 32 , wherein the formulation is administered for at least 2 weeks during the patient's pregnancy. 
     
     
         46 . The method of  claim 32 , wherein the formulation is administered for at least 6 weeks during the patient's pregnancy. 
     
     
         47 . The method of  claim 32 , wherein the formulation is administered for at least 12 weeks during the patient's pregnancy. 
     
     
         48 . The method of  claim 32 , wherein the formulation is administered during a first trimester of the patient's pregnancy. 
     
     
         49 . The method of  claim 32 , wherein the formulation is administered during a second trimester of the patient's pregnancy. 
     
     
         50 . The method of  claim 32 , wherein the formulation is administered during the first 16 weeks of the patient's pregnancy.