Galafold fachinformation
WebMay 30, 2024 · Galafold ® capsules 123 mg (migalastat) is a first-in-class chaperone therapy approved in Japan as a monotherapy for Fabry disease in patients with amenable mutations. Galafold works by stabilizing the body’s own dysfunctional enzyme, so it can clear the accumulation of disease substrate in patients who have amenable mutations. WebGALAFOLD™ is indicated for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable . alpha galactosidase A (GLA) gene variant based on . in vitro. assay data [see Clinical Pharmacology (12.1)]. This indication is approved under accelerated approval based on reduction in kidney interstitial capillary cell .
Galafold fachinformation
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Webdescribe the clinical benefit of Galafold (migalastat) in patients with Fabry disease. The trial will evaluate the efficacy and pharmacodynamic effects (e.g., on plasma lyso-Gb3, alpha-Gal A enzyme activity) of Galafold (migalastat) in patients with a confirmed diagnosis of Fabry disease and amenable, disease-causing GLA variants. The trial ... WebWhat is Galafold? Galafold is a prescription medicine used to treat adults with Fabry disease who have a certain genetic change (variant) in the galactosidase alpha gene …
WebGALAFOLD is a drug for treatment of Fabry disease in adults. It is used in patients who have a specific change in the gene that causes Fabry disease. WebAug 2, 2024 · Galafold® is the First and Only Oral Therapy Approved in the EU for the Long-term Treatment of Adolescents with Fabry Disease Aged 12 years and older with an Amenable MutationPHILADELPHIA, Aug ...
WebPrior Authorization is recommended for prescription benefit coverage of Galafold. All approvals are provided for the duration noted below. Because of the specialized skills required for evaluation and diagnosis of patients treated with Galafold as well as the monitoring required for adverse events and long-term efficacy, approval WebGalafold® 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) substrate.
WebFor those prescribed Galafold, AMICUS ASSIST is a call 1-833-AMICUS-A ( 1-833-264-2872) or click away. AMICUS ASSIST is a service designed to provide product assistance and support for Galafold. As part of the program, you’ll be assigned a dedicated case manager. * Individuals must be prescribed Galafold for an approved indication to be ...
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 … dave haskell actorWebMigalastat, sold under the brand name Galafold, is a medication for the treatment of Fabry disease, a rare genetic disorder. It was developed by Amicus Therapeutics. The US … dave harlow usgsWebrequires Galafold to be prescribed by or in consultation with a physician who specializes in the condition being treated. FDA Indication(s) 1. Fabry Disease. Approve for 1 year if the individual meets the following criteria (A, B, and C): A) Individual is ≥ … dave hatfield obituaryWebThe efficacy of Galafold was evaluated in Study AT1001-011. This study included a 6-month randomized, double-blind, placebo-controlled phase followed by a 6-month open-label treatment phase and a 12-month open-label extension phase. A total of 67 patients with Fabry disease who were naïve to Galafold and enzyme replacement therapy (ERT) … dave hathaway legendsWebJan 24, 2024 · Common Galafold side effects may include: headache; nausea; fever; or. cold symptoms such as stuffy nose, sneezing, sore throat. This is not a complete list of … dave harvey wineWebGalafold 123 mg capsule. Color: white,blue Shape: oblong Imprint: A1001 . This medicine is a white blue, oblong, capsule imprinted with "A1001". Next . You Might Also Like. dave harkey construction chelanWebWhat 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 … dave harrigan wcco radio