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Correspondence |
Bone crisis in stable Gaucher's disease
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Sir,
During the past 20 years, modern treatments have dramatically changed the prognosis for symptomatic patients with type 1 Gaucher's disease (GD). Since 1991, therapy has shifted from an approach primarily directed at symptom control to the actual reduction of glucocerebroside cellular burden. The latter therapeutic option has been realized through enzyme replacement therapy (ERT) and, more recently, with the use of miglustat, an oral substrate synthesis inhibitor (an
Hospital Clínico Universitario Zaragoza Spain
Neurogenetics Program New York University School of Medicine New York USA
email: mibh-jperezcu@lblesa.es