ArticleEpilepsy research2026
4-Phenylbutyrate rescues neurobehavioral phenotypes in SLC6A1-related encephalopathy.
Article in Epilepsy research, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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Who cites it
5 citing papers in PubMed.
- 4-Phenylbutyrate Rescue inCells · 2026Article
- Artificial intelligence in the assessment of epilepsy-related genetic mutations: Learned from GABAEpilepsia open · 2026Review
- 4-Phenylbutyrate Rescue inbioRxiv : the preprint server for biology · 2026Article
- Accumulation of Extracellular GABA, Impaired GABAergic Neurotransmission and 4-Phenylbutyrate Rescue in Mice ofbioRxiv : the preprint server for biology · 2026Article
- Molecular Basis of GABA Aminotransferase Inhibition in Epilepsy: Structure, Mechanisms, and Drug Development.Current issues in molecular biology · 2025Review
Corrections and comments
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Authors and funding
3 authors.
Funding
Abstract
Developmental and epileptic encephalopathies (DEEs) caused by pathogenic variants in SLC6A1, encoding γ-aminobutyric acid (GABA) transporter 1 (GAT-1), present with seizures and severe comorbidities including cognitive impairment, developmental delay, motor difficulties, and social deficits. Current therapies focus on seizure reduction but do not address the disabling neurobehavioral symptoms. We previously showed that 4-phenylbutyrate (PBA), known as a chemical chaperone and histone deacetylase inhibitor, restores GAT-1 function and reduces seizures in both mouse models and humans with SLC6A1 variants. Here, we tested whether PBA can improve neurobehavioral deficits in the Slc6a1
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Registered trials
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