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Fabry disease is a rare, progressive genetic disorder characterized by a defective gene (gla) that causes an enzyme deficiency [6] the european medicines agency's committee for medicinal products for human use (chmp) granted. This enzyme is responsible for breaking down disease substrate that, when deficient in patients with fabry disease, builds up in the kidneys, one of the organ systems impacted by fabry disease.
Amicus assist is a program designed to provide education, assistance, and support in gaining access to galafold to eligible patients with fabry disease and an amenable gla variant. The us food and drug administration (fda) granted it orphan drug status in 2004, [5] and the european commission followed in 2006 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
Amicus therapeutics please select the links below to view educational resources and websites provided by amicus therapeutics Community of us website fabry disease infographic A visual guide to understanding fabry disease fabry disease and hearing loss understanding and managing fatigue understanding and managing gastrointestinal symptoms Amicus therapeutics’ first oral drug for the rare condition fabry disease has been approved by the fda, giving patients an alternative to conventional injected enzyme replacement therapy.
Support made simple for your patients with fabry disease if you’ve prescribed your patient an amicus therapeutic product, we can help streamline the next steps Count on amicus assist® to provide the education and support your patients may need to get the most of their care. What is galafold for fabry disease Galafold (migalastat) is an oral chaperone therapy that’s approved for adults with fabry disease carrying certain mutations in the gla gene, the gene that’s deficient in fabry
Developed by amicus therapeutics, the therapy works to reduce the accumulation of.
Educating about fabry disease and your patient’s amicus medication providing tips to help patients establish a treatment routine helping patients have more productive conversations with their care team pels do not give medical advice or take the place of the patient’s healthcare provider. Migalastat, 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
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