Genetic & Genomic MedicineJCI InsightPeer-reviewed
A Phase 1b/2a Clinical Trial of Dantrolene Sodium in Patients with Wolfram Syndrome
Damien Abreu, Stephen Stone, Toni S. Pearson…·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.
46 citations