Therapeutic approaches to bone pathology in Gaucher disease: past, present and future.

Abstract

Enzyme replacement therapy (ERT) is effective for the treatment of the systemic manifestations of Gaucher disease (GD) and can have a significant impact on skeletal manifestations. Bone involvement is broad and can occur in otherwise clinically asymptomatic individuals. The heterogeneity in GD-related bone disease may implicate multiple pathological… (More)
DOI: 10.1016/j.ymgme.2011.08.004

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