Drug Action
Miglustat is an inhibitor of glucosylceramide synthase.
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Drug reference
Miglustat is an inhibitor of glucosylceramide synthase.
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Mild to moderate type I Gaucher’s disease for whom enzyme replacement therapy is unsuitable (under expert supervision) ▶BY MOUTH ▶Adult: 100 mg 3 times a day, reduced if not tolerated to 100 mg 1–2 times a day Treatment of progressive neurological manifestations of Niemann-Pick type C disease (under expert supervision) ▶BY MOUTH ▶Adult: 200 mg 3 times a day
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▶Common or very common Appetite decreased. asthenia. chills. constipation. depression. diarrhoea. dizziness. flatulence. gastrointestinal discomfort. headache. insomnia. libido decreased. malaise. muscle spasms. muscle weakness. nausea. peripheral neuropathy. sensation abnormal. thrombocytopenia. tremor. vomiting . weight decreased
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Effective contraception must be used during treatment. Men should avoid fathering a child during and for 3 months after treatment.
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Manufacturer advises avoid—toxicity in animal studies.
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Manufacturer advises avoid—no information available.
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Manufacturer advises caution (no information available).
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Avoid if eGFR less than 30 mL/minute/1.73 m2. Dose adjustments For Gaucher’s disease initially 100 mg twice daily if eGFR 50–70 mL/minute/1.73 m2. Initially 100 mg once daily if eGFR 30–50 mL/minute/1.73 m2. For Niemann-Pick type C disease, initially 200 mg twice daily if eGFR 50–70 mL/minute/1.73 m2. Initially 100 mg twice daily if eGFR 30–50 mL/minute/1.73 m2
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▶Monitor cognitive and neurological function. ▶Monitor growth and platelet count in Niemann-Pick type C disease.
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There can be variation in the licensing of different medicines containing the same drug. Capsule ▶Miglustat (Non-proprietary) Miglustat 100 mg Miglustat 100mg capsules | 84 capsule P £3,934.00 (Hospital only) ▶Zavesca (Actelion Pharmaceuticals UK Ltd) Miglustat 100 mg Zavesca 100mg capsules | 84 capsule P £3,934.17 (Hospital only) 2.10 Pompe disease ENZYMES
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100 mg 3 times a day, reduced if not tolerated to
First-line fragment. Read the complete Indications and Dose section above.
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200 mg 3 times a day
First-line fragment. Read the complete Indications and Dose section above.
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