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Profile of eliglustat tartrate in the management of Gaucher disease
Authors Sechi A, Dardis A, Bembi B
Received 22 July 2015
Accepted for publication 30 November 2015
Published 11 January 2016 Volume 2016:12 Pages 53—58
DOI https://doi.org/10.2147/TCRM.S73226
Checked for plagiarism Yes
Review by Single anonymous peer review
Peer reviewer comments 4
Editor who approved publication: Professor Garry Walsh
Annalisa Sechi, Andrea Dardis, Bruno Bembi
Regional Coordinator Center for Rare Diseases, Academic Hospital of Udine, Udine, Italy
Abstract: Gaucher disease (GD) is a lysosomal storage disorder caused by the deficient activity of acid beta glucosidase, with consequent accumulation of glucosylceramide in the spleen, liver, bone marrow, and various organs and tissues. Currently, the gold standard for GD treatment is enzyme replacement therapy (ERT). The efficacy of ERT in improving or stabilizing the visceral and hematological symptoms of GD is well-proven. However, since ERT has to be administered by frequent intravenous infusions, this therapeutic approach has an important impact on the patient’s quality of life. Eliglustat tartrate is a new substrate reduction therapy for GD, which acts as a specific and potent inhibitor of glucosylceramide synthase and can be administered orally. This review summarizes the results of the preclinical and clinical trials, which experimented with eliglustat, and discusses its possible role in the management of GD, when compared to the currently available treatments and the new experimental approaches.
Keywords: Gaucher disease, enzyme replacement therapy, substrate reduction therapy, eliglustat tartrate
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