A Phase 1b/2a Clinical Trial of Dantrolene Sodium in Patients with Wolfram Syndrome
Damien Abreu, Stephen Stone, Toni S. Pearson, Robert C. Bucelli, Ashley N. Simpson, Stacy Hurst, CRIS M. BROWN, Kelly Kries, Chinyere Onwumere, Hongjie Gu, James Hoekel, Lawrence Tychsen, Gregory P. Van Stavern, Neil H. White, Bess Adkins Marshall, , Fumihiko Urano
This paper reports a phase 1b/2a clinical trial of dantrolene sodium in Wolfram syndrome patients, showing it is well tolerated and showing some potential efficacy in beta-cell function and visual acuity.
JCI Insight · Jun 29, 2021Peer-reviewedGenetic & Genomic Medicine