Results 1 to 10 of about 624 (116)

Phase II trial of sodium phenylbutyrate and taurursodiol in Wolfram syndrome. [PDF]

J Clin Invest
Urano F, Marshall BA, Hurst S, Robichaux-Viehoever A, Ahmadi S, Hershey T, Van Stavern G, Cruz Bravo P, Powers Carson J, Pesko J, Fox K, Erpelding N, Bedrosian CL.   +12 more
europepmc   +1 more source

Pre-emptive treatment in later-onset urea cycle disorders: a clinical perspective on glycerol phenylbutyrate. [PDF]

Orphanet J Rare Dis
Spada M, Porta F.
europepmc   +1 more source

Parent-Reported Improvement in Seizure Control and Development After Phenylbutyrate Treatment in Children With STXBP1 and SLC6A1. [PDF]

Pediatr Neurol
Barbour K, Stӧdberg T, Larsson A, Dahlin M, Grinspan ZM.   +4 more
europepmc   +1 more source

ER Stress Regulates HIV Tat and Alcohol-Induced NLRP6 Activation in Astrocytes. [PDF]

CNS Neurosci Ther
Singh S, Horanieh E, Buch S, Periyasamy P.   +3 more
europepmc   +1 more source

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

Pharmacol Res Perspect
Frick A, Whyte-Fagundes P, Baraban SC.
europepmc   +1 more source

Real-World Clinical Experience With Sodium Phenylbutyrate and Taurursodiol at a Single Amyotrophic Lateral Sclerosis Center in the United States. [PDF]

Eur J Neurol
Calcagno N, Scirocco E, Clampffer E, Addy G, Morgan S, Parikh N, Neel DV, Scalia J, Young R, Locatelli M, Mackie DS, Berry JD, Paganoni S.   +12 more
europepmc   +1 more source

Treatment strategies, radiological recovery, and neurodevelopmental outcomes in paediatric Maple Syrup Urine Disease: a 20-year single-centre experience from Türkiye. [PDF]

Metab Brain Dis
Uylaş K, Yazıcı H, Altınok YA, Çelik MY, Karapınar FE, Özkaya PY, Isik E, Eraslan C, Canda E, Cogulu O, Karapınar B, Habif S, Çoker M, Uçar SK.   +13 more
europepmc   +1 more source

4-phenylbutyric acid-loaded mesoporous silica nanoparticles alleviate endoplasmic reticulum stress and reduce intraocular pressure in glaucoma model. [PDF]

Biol Direct
Yan X, Qin W, Gan X, Mao Y, Wu S, Wen S, Zhang J.   +6 more
europepmc   +1 more source

4-Phenylbutyrate Plus Wildtype GAT-1 Augmentation: A Dual Therapy to Rescue SLC6A1 Variant-Associated Developmental and Epileptic Encephalopathy. [PDF]

Genes (Basel)
Delahanty AJ, James K, Carter EG, Song ZD, Wang J, Bassette M, Kang JQ.   +6 more
europepmc   +1 more source

Metabolic alterations in Snyder-Robinson syndrome lymphoblasts are ameliorated by phenylbutyrate treatment. [PDF]

Mol Genet Metab
Tao X, Allen B, Wilson E, Spencer J, Norris J, Van Sickle E, Benjock J, Vickery Z, Chen CF, Skinner C, Schwartz CE, Zhai RG, Boccuto L.   +12 more
europepmc   +1 more source