Article
Neuronopathic Gaucher disease: demographic and clinical features of 131 patients enrolled in the International Collaborative Gaucher Group Neurological Outcomes Subregistry.
Journal of inherited metabolic disease - 1 Aug 2010
Tylki-Szymańska Anna, Vellodi Ashok, El-Beshlawy Amal, Cole J Alexander, Kolodny Edwin
Abstract excerpt
OBJECTIVE: To describe demographic, genetic, and clinical characteristics of patients with neuronopathic Gaucher disease (NGD). METHODS: All patients enrolled in the Neurological Outcomes Subregistry of the International Collaborative Gaucher Group (ICGG) Gaucher Registry as of June 2007 were identified. RESULTS: The study cohort comprised 131 patients from 17 countries who were enrolled in the Neurological...
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