What is Galafold and how does it work?
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.
What are the side effects of Galafold?
The most common side effects of Galafold include:
These are not all the possible side effects of Galafold. Call your doctor 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 1-877-426-4287.
What is the dosage for Galafold?
- The recommended dosage regimen of Galafold is 123 mg orally once every other day at the same time of day.
- Take Galafold on an empty stomach. Do not consume food at least 2 hours before and 2 hours after taking Galafold to give a minimum 4 hours fast. Clear liquids can be consumed during this 4-hour period.
- Do not take Galafold on 2 consecutive days.
- If a dose is missed entirely for the day, take the missed dose of Galafold only if it is within 12 hours of the normal time that the dose should have been taken. If more than 12 hours have passed, resume taking Galafold at the next planned dosing day and time, according to the every-other-day dosing schedule.
- Swallow capsules whole. Do not cut, crush, or chew.
Is Galafold safe to take when pregnant or breastfeeding?
- The estimated background risk for major birth defects and miscarriage for the indicated population is unknown.
- There is a registry that monitors outcomes in individuals with Fabry disease, either exposed or unexposed to Galafold during pregnancy and/or while breastfeeding infants up to 1 year of age.
- Healthcare providers are encouraged to register patients or obtain additional information by contacting the Pregnancy Coordinating Center at 1-888-239-0758, email at [email protected], or visit www.fabrypregnancyregistry.com.
- There are no human data available on the presence of migalastat in human milk, the effects on the breastfed infant, or the effects on milk production.
- The developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for Galafold and any potential adverse effects on the breastfed child from Galafold or from the underlying maternal condition.
Galafold (migalastat) 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.
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Related Disease Conditions
Fabry disease (Fabry's disease, alpha-galactosidase-A) is a genetic disorder with symptoms such as burning sensations in the hands, small-raised reddish-purplish blemishes on the skin, fever, decreases sweating, and gastrointestinal (GI) difficulties. Fabry disease patients are at increased risk of heart attack, heart disease, kidney failure, and stroke. Symptoms of Fabry disease can be treated with medication.
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The definition of a genetic disease is a disorder or condition caused by abnormalities in a person's genome. Some types of genetic inheritance include single inheritance, including cystic fibrosis, sickle cell anemia, Marfan syndrome, and hemochromatosis. Other types of genetic diseases include multifactorial inheritance. Still other types of genetic diseases include chromosome abnormalities (for example, Turner syndrome, and Klinefelter syndrome), and mitochondrial inheritance (for example, epilepsy and dementia).
Your health care provider may refer you to a genetic professional. Universities and medical centers also often have affiliated genetic professionals, or can provide referrals to a genetic professional or genetics clinic. Genetic counseling provides patients and family members the tools to make the right choice in regard to test for a disease or condition.
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In the nature vs. nurture debate, "nature" represents our genetic makeup. These are the genes you have inherited from your biological family, and that may affect your physical and mental health, for example, intelligence, disease, and psychological health. While "nurture" represents how our environment affects our intelligence, traits, personality, and mental and physical health. Studies have shown that a person's environment can alter his or her genes, and lower their risk of developing certain inherited diseases, conditions, and mental illnesses that run in his or her family. Researchers and doctors have found that particular physical traits like eye and skin color, and diseases like Huntington's chorea are the result of genetic inheritance (inherited from a family member). However, patterns of thinking and behavior can be attributed to both nature and nurture (your genes and your environment). Moreover, researchers who study the brain have found overwhelming evidence that a person's genetic factors and his or her experiences guide and support brain development. The human brain produces new nerve cells (neurons) into adulthood, and these nerve cells can change the strength of their connections throughout life, which can affect intelligence and other factors.
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