How does galafold work for fabry disease Fabry disease is a rare inherited condition that is passed down from parents to their children. Galafold ® (migalastat) is indicated for the treatment of adults with a confirmed diagnosis of fabry disease and an amenable galactosidase alpha gene () variant based on assay data Continued approval for this indication may be contingent. Galafold ® (migalastat) is an oral prescription medicine indicated for the treatment of adults with fabry disease who have a responsive galactosidase alpha (gla) gene variant, as determined by in vitro assay data 1 developed by amicus therapeutics, 2 the therapy was approved by the us food and drug administration (fda) in august 2018
3 fabry disease is a rare, inherited, multisystemic. Galafold is an approved oral therapy for adults with fabry disease and amenable mutations that helps to clear cells of toxic fatty molecules. Galafold ® is indicated for the treatment of adults with a confirmed diagnosis of fabry disease and an amenable galactosidase alpha gene (gla) variant based on in vitro assay data Galafold is indicated for the treatment of adults with a confirmed diagnosis of fabry disease and an amenable galactosidase alpha gene (gla) variant based on in vitro assay data Galafold contains the active substance migalastat Galafold is a prescription medicine used to treat adults with fabry disease who have a certain genetic change (variant) in the galactosidase alpha gene (gla) that is responsive (amenable) to galafold
