About Fabry
> Disease Overview What is Fabry disease?

Alex, an actual Fabry patient.

What is Fabry disease?

Fabry is a rare disease caused by changes in the GLA gene, which may be referred to as mutations or variants.

People with Fabry disease have trouble breaking down and getting rid of certain fatty waste substances (substrates) in cells. This happens because of a variant in the GLA gene that leads to a deficient or absent enzyme called alpha-Gal A. When alpha-Gal A is present and functioning, it travels to the cell’s recycling center, called the lysosome, to break down the fatty substances in the cell.

MOD | Properly Folded Alpha Gal-A

In Fabry disease, without fully functioning alpha-Gal A, the fatty substances build up in the body, causing damage to tissues and organs.

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Explore the symptoms of Fabry disease

Fabry disease has a wide range of symptoms that may vary from person to person.

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IMPORTANT SAFETY INFORMATION AND INDICATION

Before taking Galafold® (migalastat), tell your healthcare provider about all of your medical conditions, including if you:

  • have kidney problems.
  • are pregnant or plan to become pregnant. It is not known if Galafold will harm your unborn baby.
  • have kidney problems.
  • are pregnant or plan to become pregnant. It is not known if Galafold will harm your unborn baby.
  • are breastfeeding or plan to breastfeed. Galafold may pass into breast milk. Talk to your healthcare provider
    about the best way to feed your baby if you take Galafold.

Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Especially tell your healthcare provider if you take medicines or supplements containing caffeine as these medicines or supplements may affect how Galafold works.

How should I take Galafold?

Do not eat food, or take or drink any product that contains caffeine at least 2 hours before and 2 hours after taking Galafold to give a minimum 4 hour fast.

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.

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.

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.

Please click here for Full Prescribing Information, including Patient Information and Instructions for Use.

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