| Title |
Profile of eliglustat tartrate in the management of Gaucher disease
|
|---|---|
| Published in |
Therapeutics and Clinical Risk Management, January 2016
|
| DOI | 10.2147/tcrm.s73226 |
| Pubmed ID | |
| Authors |
Annalisa Sechi, Andrea Dardis, Bruno Bembi |
| 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. |
X Demographics
The data shown below were collected from the profile of 1 X user who shared this research output. Click here to find out more about how the information was compiled.
Geographical breakdown
| Country | Count | As % |
|---|---|---|
| United States | 1 | 100% |
Demographic breakdown
| Type | Count | As % |
|---|---|---|
| Scientists | 1 | 100% |
Mendeley readers
The data shown below were compiled from readership statistics for 34 Mendeley readers of this research output. Click here to see the associated Mendeley record.
Geographical breakdown
| Country | Count | As % |
|---|---|---|
| Unknown | 34 | 100% |
Demographic breakdown
| Readers by professional status | Count | As % |
|---|---|---|
| Researcher | 10 | 29% |
| Other | 4 | 12% |
| Student > Master | 4 | 12% |
| Student > Ph. D. Student | 3 | 9% |
| Student > Bachelor | 3 | 9% |
| Other | 2 | 6% |
| Unknown | 8 | 24% |
| Readers by discipline | Count | As % |
|---|---|---|
| Medicine and Dentistry | 10 | 29% |
| Biochemistry, Genetics and Molecular Biology | 5 | 15% |
| Pharmacology, Toxicology and Pharmaceutical Science | 4 | 12% |
| Agricultural and Biological Sciences | 4 | 12% |
| Chemistry | 2 | 6% |
| Other | 2 | 6% |
| Unknown | 7 | 21% |
Attention Score in Context
This research output has an Altmetric Attention Score of 1. This is our high-level measure of the quality and quantity of online attention that it has received. This Attention Score, as well as the ranking and number of research outputs shown below, was calculated when the research output was last mentioned on 27 January 2016.
All research outputs
#20,657,128
of 25,374,917 outputs
Outputs from Therapeutics and Clinical Risk Management
#1,070
of 1,323 outputs
Outputs of similar age
#295,044
of 399,677 outputs
Outputs of similar age from Therapeutics and Clinical Risk Management
#52
of 55 outputs
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