An open-label, noncomparative study of miglustat in type I Gaucher disease: Efficacy and tolerability over 24 months of treatment

In this small study in symptomatic adult patients with type I GD, miglustat treatment resulted in a significant decrease in liver and spleen volume at 6 and 18 months, with clinical improvement noted over 24 months. Bone involvement and platelet and hemoglobin values remained stable, with no significant changes noted during the observation period. The effects of treatment were consistent with those of earlier studies of miglustat in type I GD.