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Article

Bicyclic derivatives of L-idonojirimycin as pharmacological chaperones for neuronopathic forms of Gaucher disease.

Chembiochem : a European journal of chemical biology - 27 May 2013

Alfonso Pilar, Andreu Vanesa, Pino-Angeles Almudena, Moya-García Aurelio A, García-Moreno M Isabel, Rodríguez-Rey José C, Sánchez-Jiménez Francisca, Pocoví Miguel, Ortiz Mellet Carmen, García Fernández Jose M, Giraldo Pilar

Abstract excerpt

New human β-glucocerebrosidase (GCase) ligands with rigid 1,6-anhydro-β-L-idonojirimycin cores have been designed with the aid of molecular modeling. Efficient pharmacological chaperones for the L444P (trafficking-incompetent) mutant GCase enzyme associated with type 2 and 3 Gaucher disease (GD)...

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Identifiers

PMID: 23606264
DOI: 10.1002/cbic.201200708

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 4 Sept 2026. Prepared record publication-23606264-2026-09-04.2. Manifest publication-graph-cohort-2026-09-04.2.

Source receipt: PubMed via Helena Literature Corpus, retrieved 4 Sept 2026.

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