Results for tag: "Galafold"

Galafold (migalastat) is a drug used to treat Fabry disease, a rare, inherited endocrine and metabolic disorder. It is a pharmacological chaperone, meaning it binds to the mutated enzyme and helps it to fold correctly, allowing it to function normally. Galafold is the first and only approved pharmacological chaperone for Fabry disease. It is taken orally, and is designed to reduce the amount of globotriaosylceramide (GL-3) in the body, which is the cause of the disease. Galafold is a relatively new drug, having been approved by the FDA in 2018. It is currently marketed by Amicus Therapeutics, a biopharmaceutical company based in the United States. Other companies in the market include Shire, Sanofi, and Genzyme. Show Less Read more