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.
The primary objective was to assess the safety and tolerability of dantrolene sodium in adult and pediatric Wolfram syndrome patients. Secondary objectives were to evaluate the efficacy of dantrolene sodium on residual pancreatic beta cell functions, visual acuity, …
The primary objective was to assess the safety and tolerability of dantrolene sodium in adult and pediatric Wolfram syndrome patients …