Effect of miglustat on bone disease in adults with type 1 Gaucher disease: a pooled analysis of three multinational, open-label studies
GM Pastores, D Elstein, M Hrebícek, A Zimran - Clinical therapeutics, 2007 - Elsevier
Background: Bone manifestations are a source of disability among patients with Gaucher
disease (GD) and a focus of disease management. The effect of enzyme replacement
therapy (ERT) on GD bone disease can be limited and may take up to 8 years to become
manifest. Miglustat, a glucosylceramide synthase inhibitor, may have a positive influence on
GD bone disease. Objectives: The aim of this analysis was to evaluate the effects of
miglustat on bone manifestations and bone mineral density (BMD) in patients with type 1 …
disease (GD) and a focus of disease management. The effect of enzyme replacement
therapy (ERT) on GD bone disease can be limited and may take up to 8 years to become
manifest. Miglustat, a glucosylceramide synthase inhibitor, may have a positive influence on
GD bone disease. Objectives: The aim of this analysis was to evaluate the effects of
miglustat on bone manifestations and bone mineral density (BMD) in patients with type 1 …