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Imiglucerase in the treatment of Gaucher disease: a history and perspective

Authors Deegan PB, Cox T

Received 14 November 2011

Accepted for publication 15 December 2011

Published 18 April 2012 Volume 2012:6 Pages 81—106

DOI https://doi.org/10.2147/DDDT.S14395

Review by Single-blind

Peer reviewer comments 4


Patrick B Deegan, Timothy M Cox
Department of Medicine, University of Cambridge, Lysosomal Disorders Unit, Addenbrooke's NHS Foundation Hospitals Trust, Cambridge, UK

Abstract: The scientific and therapeutic development of imiglucerase (Cerezyme®) by the Genzyme Corporation is a paradigm case for a critical examination of current trends in biotechnology. In this article the authors argue that contemporary interest in treatments for rare diseases by major pharmaceutical companies stems in large part from an exception among rarities: the astonishing commercial success of Cerezyme. The fortunes of the Genzyme Corporation, latterly acquired by global giant Sanofi SA, were founded on the evolution of a blockbuster therapy for a single but, as it turns out, propitious ultra-orphan disorder: Gaucher disease.

Keywords: enzyme therapy, ultra-orphan, macrophage targeting, lysosomal disease, mannose lectin, biopharmaceutical

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