Zavesca® (miglustat) is indicated for the treatment of adult patients with mild to moderate type 1 Gaucher disease for whom enzyme replacement therapy is not a therapeutic option (e.g., due to constraints such as allergy, hypersensitivity, or poor venous access).
In clinical studies, the most common adverse events due to Zavesca included weight loss, diarrhea, and trembling in the hand (tremor). Other common adverse reactions were excess gas (flatulence), abdominal pain, headache, and influenza-like symptoms. The most common serious adverse reaction was tingling or numbness in the hands or feet with or without pain (peripheral neuropathy). Patients should undergo neurological examination at the start of treatment and every 6 months thereafter; Zavesca should be reassessed in patients who develop symptoms of peripheral neuropathy. Zavesca may cause fetal harm if administered to a pregnant woman. Men should maintain reliable contraceptive methods and not plan to father a child while taking Zavesca and for 3 months after discontinuing treatment.
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