Phase 2a/b randomised placebo-controlled dose-escalation trial of triheptanoin for ataxia-telangiectasia: treating mitochondrial dysfunction with anaplerosis.

Statistics Based on our in vitro data showing a highly significant difference (p < 0.001) in cell killing between A-T and control cells and correction with heptanoate, we simulated data assuming 60%, 50%, 40% and 25% cell death in individuals receiving 0%, 10%, 20%, 35% doses respectively, with standard deviation (SD) = 15%.

also in 132,142 other papers