WebWhat is Galafold? Galafold® (migalastat) is a prescription medicine used to treat adults with Fabry disease who have a certain genetic change (variant) in the galactosidase … WebSep 17, 2024 · Galafold is a medicine used to treat patients aged 12 years and above who have Fabry disease. This is a rare inherited disorder where patients have various …
Galafold™ (migalastat) Referral Form - Accredo
WebOct 1, 2024 · In an earlier Galafold phase 3 study in 67 patients, the FACETS trial, treatment-naive patients receiving the Amicus small molecule for 12 months had durable reductions in levels of kidney ... WebGalafold ® (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. This indication is approved under accelerated approval based on reduction in kidney interstitial capillary cell globotriaosylceramide (KIC GL-3 ... cleethorpes to derby train
Galafold: Cost, usage, side effects, how it works, and more
WebJul 12, 2024 · Morbus Fabry, eine seltene lysosomale Speichererkrankung, wird durch eine Mutation im GLA-Gen hervorgerufen, die zu einer Fehlfunktion des Enzyms Alpha-Galaktosidase A führt. Die Erkrankung ist durch vielfältige Symptome wie neuropathische brennende Schmerzen an Händen und Füssen, Angiokeratome, frühe Schlaganfälle, … WebGalafold ® (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 … WebINDICATIONS AND USAGE. 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 … cleethorpes to grimsby