Press Announcements > FDA approves new drug to treat a form of Gaucher disease

Press Announcements > FDA approves new drug to treat a form of Gaucher disease

Gaucher Disease: Progress in Therapy for Rare Disease

FDA approves new drug to treat a form of Gaucher disease, FDA, Aug 19

What is Gaucher disease? From the NIH Genetics Home Reference

Find out what genetic conditions and tests are associated with Gaucher disease, from the NIH Genetic Testing Registry

Early diagnosis of Gaucher disease in pediatric patients: Proposal for a diagnostic algorithm. 
Di Rocco M et al. Pediatr Blood Cancer. 2014 Aug 17

Long term effects of enzyme replacement therapy in an Italian cohort of type 3 Gaucher patients. 
Sechi A, et al. Mol Genet Metab. 2014 Aug 4

Modelling Gaucher disease progression: long-term enzyme replacement therapy reduces the incidence of splenectomy and bone complications. 
van Dussen L, et al. Orphanet J Rare Dis. 2014 Jul 24;9(1):112.

Find more information about recent genomic insights in rare diseases from our June 11 Update page