Miglustat reduces the formation of a certain protein in the body in people with type 1 Gaucher disease.
Gaucher disease is a genetic condition in which the body lacks the enzyme needed to break down certain fatty materials (lipids). Lipids can build up in the body, causing symptoms such as easy bruising or bleeding, weakness, anemia, bone or joint pain, enlarged liver or spleen, or weakened bones that are easily fractured.
Miglustat is used to treat mild to moderate type 1 Gaucher disease by people who cannot receive enzyme replacement therapy.
Black, tarry stools
blood in the urine or stools
burning, crawling, itching, numbness, prickling, "pins and needles", or tingling feelings
pinpoint red spots on the skin
trembling or shaking in the hands
unsteadiness or awkwardness
unusual bleeding or bruising
weakness in the arms, hands, legs, or feet
Some of the side effects that can occur with miglustat may not need medical attention. As your body adjusts to the medicine during treatment these side effects may go away. Your health care professional may also be able to tell you about ways to reduce or prevent some of these side effects. If any of the following side effects continue, are bothersome or if you have any questions about them, check with your health care professional:
bloated or full feeling
blurred or loss of vision
difficulty having a bowel movement (stool)
disturbed color perception
excess air or gas in the stomach or intestines
halos around lights
heaviness in the limbs
loss of appetite
pain or discomfort in the chest, upper stomach, or throat
stomach discomfort, upset or pain
swelling of the abdominal or stomach area