Testing effectiveness (Phase 2)Ended earlyNCT04059406
What this trial is testing
Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx)
Who this might be right for
Beta Thalassemia Intermedia
Ionis Pharmaceuticals, Inc. 29