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New therapies in the management of Niemann-Pick type C disease: clinical utility of miglustat
Therapeutics and Clinical Risk Management, 11/05/09

Wraith JE et al. – Findings demonstrated clinically relevant beneficial effects of miglustat on neurological disease progression in adult, juvenile and pediatric patients with NP–C, particularly those diagnosed in late childhood and in juveniles and adults, compared with those diagnosed in early childhood. With the approval of miglustat, treatment of patients with NP–C can now be aimed toward stabilizing neurological disease, which is likely the best attainable therapeutic goal for this disorder.

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