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.
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.
Explore how Galafold acts as a pharmacological chaperone below:
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 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.”
“I found out about a possible treatment that was in development. I was told there would be a trial starting soon, and they needed to run some tests to see if I fit the criteria for their study....It turned out that my variant was amenable for the treatment. Later, that treatment was approved as Galafold and I've been on it ever since.”
Download the printable Galafold brochure to learn more.Download now
Before taking Galafold® (migalastat), tell your healthcare provider about all of your medical conditions, including if you:
Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.
What are the possible side effects of Galafold?
The most common side effects of Galafold include headache, stuffy or runny nose and sore throat, urinary tract infection, nausea, and fever.
These are not all the possible side effects of Galafold. Call your healthcare provider for medical advice about side effects.
What 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 (GLA) that is responsive (amenable) to Galafold.
It is not known if Galafold is safe and effective in children.
You may report side effects to FDA at 1-800-FDA-1088. You may also report side effects to Amicus Therapeutics at 1-877-4AMICUS.
Please click here for Full Prescribing Information, including Patient Information and Instructions for Use.