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Promoting awareness, pioneering research and providing support and information for those affected by Gaucher disease.

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Cerdelga Update

17 September 2014

Following the Food and Drug Administration (FDA) approval of Cerdelga as an oral treatment for certain adult Gaucher disease type 1 patients in August, press reports suggest that Genzyme has set a price for the US patients at $310,250 a year, see press release:  http://www.bostonglobe.com/business/2014/09/02/new-genzyme-pill-treat-rare-gaucher-disease-will-cost-patients-year/5thkIb587nKi7zRAb9GgxM/story.html

In Europe, marketing applications for Cerdelga are under review by the European Medicines Agency (EMA) and other regulatory authority, a decision is expected at the end of 2014.