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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. It is not known if galafold is safe and effective in children How does galafold work for fabry disease

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Fabry disease is a rare inherited condition that is passed down from parents to their children. 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 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