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Article

Pharmacological chaperone therapy for the treatment of inborn errors of metabolism

Postepy biochemii - 30 Sept 2022

Lipiński Patryk, Jezela-Stanek Aleksandra, Tylki-Szymańska Anna

Abstract excerpt

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. W artykule scharakteryzowano mechanizm działania chaperonów molekularnych i...

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Identifiers

PMID: 36317990
DOI: 10.18388/pb.2021_451

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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