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A phase Ib/IIa clinical trial of dantrolene sodium in patients with Wolfram syndrome.
Abreu D, Stone SI, Pearson TS, Bucelli RC, Simpson AN, Hurst S, Brown CM, Kries K, Onwumere C, Gu H, Hoekel J, Tychsen L, Van Stavern GP, White NH, Marshall BA, Hershey T, Urano F. Abreu D, et al. JCI Insight. 2021 Aug 9;6(15):e145188. doi: 10.1172/jci.insight.145188. JCI Insight. 2021. PMID: 34185708 Free PMC article. Clinical Trial.
GLP-1R agonists demonstrate potential to treat Wolfram syndrome in human preclinical models.
Gorgogietas V, Rajaei B, Heeyoung C, Santacreu BJ, Marín-Cañas S, Salpea P, Sawatani T, Musuaya A, Arroyo MN, Moreno-Castro C, Benabdallah K, Demarez C, Toivonen S, Cosentino C, Pachera N, Lytrivi M, Cai Y, Carnel L, Brown C, Urano F, Marchetti P, Gilon P, Eizirik DL, Cnop M, Igoillo-Esteve M. Gorgogietas V, et al. Diabetologia. 2023 Jul;66(7):1306-1321. doi: 10.1007/s00125-023-05905-8. Epub 2023 Mar 30. Diabetologia. 2023. PMID: 36995380 Free PMC article.
Current Landscape of Treatments for Wolfram Syndrome.
Abreu D, Urano F. Abreu D, et al. Trends Pharmacol Sci. 2019 Oct;40(10):711-714. doi: 10.1016/j.tips.2019.07.011. Epub 2019 Aug 13. Trends Pharmacol Sci. 2019. PMID: 31420094 Free PMC article. Review.
Genotype and Clinical Characteristics of Patients with Wolfram Syndrome and WFS1-related Disorders.
Lee EM, Verma M, Palaniappan N, Pope EM, Lee S, Blacher L, Neerumalla P, An W, Campbell T, Brown C, Hurst S, Marshall B, Hershey T, Nunes V, de Heredia ML, Urano F. Lee EM, et al. medRxiv [Preprint]. 2023 Feb 16:2023.02.15.23284904. doi: 10.1101/2023.02.15.23284904. medRxiv. 2023. Update in: Front Genet. 2023 Jun 21;14:1198171. doi: 10.3389/fgene.2023.1198171 PMID: 36824811 Free PMC article. Updated. Preprint.
14 results