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Virtual Screening and Zebrafish Phenotype-Based Evaluation Argues Against Repurposing 4-Phenylbutyrate for STXBP1-Related Disorders.

Pharmacology research & perspectives - 1 Jun 2026

Frick Aline, Whyte-Fagundes Paige, Baraban Scott C

Abstract excerpt

Syntaxin-binding protein 1 (STXBP1) mutations lead to severe epilepsy, intellectual disability, developmental delay, and movement disorder. Effective treatments for these conditions do not exist. Recent studies in Munc18-1 (STXBP1) C. elegans models demonstrate that 4-phenylbutyrate (4-PBA) or related pharmacological chaperones stabilize Munc18-1 protein levels and rescue locomotion deficits. These studies...

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Disease Models, Animal
Drug Repositioning
Epilepsy
Locomotion
Munc18 Proteins

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Phenylbutyrates

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Open in PubMed Open full text

Identifiers

PMID: 42268630
DOI: 10.1002/prp2.70285
PMCID: PMC13251787

Genes

STXBP1

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 1 Sept 2026.

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Virtual Screening and Zebrafish Phenotype-Based Evaluation Argues Against Repurposing 4-Phenylbutyrate for STXBP1-Related Disorders.PMID 42268630Read ↗

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