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Article

5-C-Branched Deoxynojirimycin: Strategy for Designing a 1-Deoxynojirimycin-Based Pharmacological Chaperone with a Nanomolar Affinity for Pompe Disease.

Journal of medicinal chemistry - 10 Feb 2022

Kato Atsushi, Nakagome Izumi, Kanekiyo Uta, Lu Tian-Tian, Li Yi-Xian, Yoshimura Kosuke, Kishida Mana, Shinzawa Kenta, Yoshida Tomoki, Tanaka Nobutada, Jia Yue-Mei, Nash Robert J, Fleet George W J, Yu Chu-Yi

Abstract excerpt

In recent years, the function of pharmacological chaperones as a "thermodynamic stabilizer" has been attracting attention in combination therapy. The coadministration of a pharmacological chaperone and recombinant human acid α-glucosidase (rhGAA) leads to improved stability and maturation by binding to the folded state of the rhGAA and thereby promotes enzyme delivery. This study provides the first example of a...

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Identifiers

PMID: 35072486
DOI: 10.1021/acs.jmedchem.1c01673

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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