Patryk Lipiński, Aleksandra Jezela-Stanek, Anna Tylki-Szymańska
{"title":"Pharmacological chaperone therapy for the treatment of inborn errors of metabolism","authors":"Patryk Lipiński, Aleksandra Jezela-Stanek, Anna Tylki-Szymańska","doi":"10.18388/pb.2021_451","DOIUrl":null,"url":null,"abstract":"<p><p>The article describes the mechanism of molecular and pharmacological chaperones in the treatment of inborn errors of metabolism. The literature review of the usage of ambroxol acting as a pharmacological chaperone for beta-glucocerebrosidase in Gaucher disease and Parkinson’s disease associated with GBA variants has been reviewed.</p>","PeriodicalId":20335,"journal":{"name":"Postepy biochemii","volume":"68 3","pages":"255-263"},"PeriodicalIF":0.0000,"publicationDate":"2022-07-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Postepy biochemii","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.18388/pb.2021_451","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2022/9/30 0:00:00","PubModel":"Print","JCR":"Q3","JCRName":"Medicine","Score":null,"Total":0}
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Abstract
The article describes the mechanism of molecular and pharmacological chaperones in the treatment of inborn errors of metabolism. The literature review of the usage of ambroxol acting as a pharmacological chaperone for beta-glucocerebrosidase in Gaucher disease and Parkinson’s disease associated with GBA variants has been reviewed.
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