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[Comment] Eliglustat for Gaucher’s disease: trippingly on the tongue

Gaucher’s disease is a rare inherited disorder resulting from a deficiency of β glucosidase and accumulation of substrate glucosyl ceramide; patients in the UK are identified once they have become symptomatic, although often a delay occurs from onset of clinical manifestations to diagnosis.1 Intravenous enzyme replacement therapy (ERT) is the mainstay of Gaucher’s disease treatment, introduced in the 1990s. Three distinct recombinant preparations are currently available—two preparations approved in Europe and three by the Food and Drug Administration in the USA.

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