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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. Among authors: kries k. JCI Insight. 2021 Aug 9;6(15):e145188. doi: 10.1172/jci.insight.145188. JCI Insight. 2021. PMID: 34185708 Free PMC article. Clinical Trial.
Multidimensional analysis and therapeutic development using patient iPSC-derived disease models of Wolfram syndrome.
Kitamura RA, Maxwell KG, Ye W, Kries K, Brown CM, Augsornworawat P, Hirsch Y, Johansson MM, Weiden T, Ekstein J, Cohen J, Klee J, Leslie K, Simeonov A, Henderson MJ, Millman JR, Urano F. Kitamura RA, et al. Among authors: kries k. JCI Insight. 2022 Sep 22;7(18):e156549. doi: 10.1172/jci.insight.156549. JCI Insight. 2022. PMID: 36134655 Free PMC article.