A Synthetic Review of Eliglustat, a Drug for Gaucher’s Disease

Gaucher’s disease (GD) is a rare genetic disorder that falls under the category of lysosomal storage diseases (LSDs) and is caused by the defective production of the lysosomal enzyme glucocerebrosidase. As a result, glucosylceramide (GlcCer) is not hydrolyzed into glucose and ceramide, which causes...

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Veröffentlicht in:Organic process research & development 2023-11, Vol.27 (11), p.1900-1912
Hauptverfasser: Pandya, Vikrant H., Kalariya, Ravi N., Bhosale, Rajesh S., Yadav, Jhillu Singh
Format: Artikel
Sprache:eng
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Zusammenfassung:Gaucher’s disease (GD) is a rare genetic disorder that falls under the category of lysosomal storage diseases (LSDs) and is caused by the defective production of the lysosomal enzyme glucocerebrosidase. As a result, glucosylceramide (GlcCer) is not hydrolyzed into glucose and ceramide, which causes GlcCer to build up in the macrophages. Births with GD are recorded at a rate of 1/40,000 to 1/60,000 in the general population, but in the case of the Ashkenazi Jewish community, this rate goes up to 1/800 births. The first oral medication for the treatment of GD was miglustate. Then a new oral medication, eliglustat (1), was introduced to treat GD type 1. In this review, we explore various synthetic approaches to synthesizing 1, which will assist in designing newer approaches.
ISSN:1083-6160
1520-586X
DOI:10.1021/acs.oprd.3c00190