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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. Galafold ® (migalastat) 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 ® (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
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Continued approval for this indication may be contingent. People with fabry disease have trouble breaking down and getting rid of certain fatty waste substances (substrates) in cells How does galafold work for fabry disease
Fabry disease is a rare inherited condition that is passed down from parents to their children.
Galafold is an approved oral therapy for adults with fabry disease and amenable mutations that helps to clear cells of toxic fatty molecules. 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.
Fabry disease is a progressive, multisystemic disease 1 that can manifest in a multitude of ways due to its impact on a range of cells and organs throughout the body 2 in the classic phenotype, neurological symptoms such as pain, abnormal tingling and numbness sensation especially in the fingers or toes and dermatological conditions such as angiokeratomas (reddish. Migalastat (galafold™), a pharmacological chaperone, stabilizes and facilitates trafficking of. Fabry is a rare disease caused by changes in the gla gene, which may be referred to as mutations or variants
