For adults with an amenable GLA variant, Galafold is designed to work with your own enzyme
What does it mean to have an amenable GLA gene variant?
Certain GLA gene variants lead to a deficient, misfolded alpha-Gal A enzyme that may be helped by Galafold. These variants are considered to be amenable.
Galafold uses the enzyme you already have. It isn’t enzyme replacement therapy (ERT).
In order to break down fatty substances, alpha-Gal A needs to travel into the lysosome. But usually only properly folded enzymes make it into this part of the cell. Misfolded enzymes, like some of those seen in Fabry disease, cannot enter the lysosome and are broken down instead. For adults with an amenable gene variant, that’s where Galafold may help.
Watch how Galafold acts as a pharmacological chaperone:
Expand to click through the individual steps
Many variants can cause Fabry disease. Galafold may work for yours.
Even if you’ve had a test in the past that determined you have little or no enzyme activity, your variant may still be amenable to Galafold. It just means your enzyme can’t fold properly and get to the lysosome to do its job.
It’s important to talk to your healthcare provider to see if Galafold may be right for you.
“I got connected with a wonderful genetic counselor. She sent me paperwork to get me and my son genetically tested.” – Rebecca, on her and her son’s variant testing.
Watch her full story >
Learn more about what it’s like taking Galafold from the How-to-Take Brochure