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miglustat (mi-gloo-stat)



Therapeutic: none assigned

Pharmacologic: enzyme inhibitors (D-glucose analogue)


Mild-to-moderate type 1 Gaucher's disease when enzyme replacement therapy is not an option.


Competitively and reversibly inhibits glucosylceramide synthase, which is the initial step in the production of glycosphingolipids. The glycosphingolipid glucosylceramide accumulates in tissues in Gaucher's disease.

Therapeutic Effects: Decreased production/accumulation of glycosphingolipid glucosylceramide with decreased tissue damage.

Adverse Reactions/Side Effects

CNS: headache. GI: abdominal pain, diarrhea, flatulence, nausea, weight loss, anorexia, dyspepsia. GU: ↓ male fertility. Hemat: thrombocytopenia. Metab: weight loss. Neuro: paresthesia, peripheral neuropathy, tremor.


Examination and Evaluation

  • Be alert for signs of paresthesias and peripheral neuropathy, as indicated by numbness, tingling, and decreased muscle strength. Establish baseline electroneuromyographic values using EMG and nerve conduction at the beginning of drug treatment whenever possible, and reexamine these values periodically to document drug-induced changes in peripheral nerve function.

  • Monitor signs of thrombocytopenia such as bruising, nose bleeds, and bleeding gums. Report these signs to the physician.

  • Periodically assess body weight and other anthropometric measures (body mass index, body composition). Report a rapid or unexplained weight loss or decreased body fat.


  • Implement therapeutic exercises (resistive exercises, gait training) to complement the effects of drug therapy and help maintain bone density.

Patient/Client-Related Instruction

  • Instruct patient to report other troublesome side effects, including prolonged or severe headache, tremor, or GI problems such as nausea, diarrhea, indigestion, loss of appetite, abdominal pain, and flatulence.


Absorption: Well absorbed following oral administration.

Distribution: Distributes into extravascular tissues.

Metabolism and Excretion: Not metabolized; excreted mostly unchanged in urine.

Half-life: 6–7 hr.

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TIME/ACTION PROFILE (blood levels)

PO unknown 2–2.5 hr 8 hr


Contraindicated in: Hypersensitivity; OB/Lactation: Pregnancy or lactation; Severe renal impairment (<30 mL/min).

Use Cautiously in: Mild-to-moderate renal impairment (dose alteration recommended if CCr <70 mL/min); Geri: Consider age related decrease in body mass, cardiac, renal and hepatic function, other chronic illnesses and concurrent drug therapies; Pedi: Children <18 yr (safety not established).


Drug-Drug: ↑ clearance of imiglucerase (should not be used concurrently).


PO (Adults): 100 mg tid at regular intervals.

Renal Impairment

PO (Adults): CCr 50–70 mL/min—100 mg bid; ...

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