Illuminating Gaucher disease

23 October 2018

By Dr Nicola Davies

Welcome again to The Pharma Letter’s new column, RARE: Illuminating Orphan Diseases. October 1 was International Gaucher Day, a day to raise awareness of a lysosomal storage disorder that annually affects one in every 60,000 individuals in the general population and one in every 1,000 individuals in the Ashkenazi Jewish population.1 We look at the current status of Gaucher disease diagnosis and treatment, as well what pharma need to do to advance the options available to this population.

What is Gaucher disease?

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