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

Drug Design, Development and Therapy
Patrick B Deegan, Timothy M Cox

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.

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Citations

Feb 13, 2013·American Journal of Hematology·Timothy M Cox
May 20, 2015·Best Practice & Research. Clinical Endocrinology & Metabolism·Timothy M Cox
Nov 6, 2020·Progress in Neurobiology·Yael Pewzner-JungAnthony H Futerman

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Methods Mentioned

BETA
electron microscopy
X-ray
biopsy
glycosylation
chemical modification

Software Mentioned

Ceredase

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