Edison's EPI-743 the first to show disease reversal promise in Leigh's
This article was originally published in Scrip
Executive Summary
All 10 children in a Phase IIa clinical trial experienced a reversal in the progression of Leigh syndrome (subacute necrotising encephalomyelopathy) during treatment with Edison Pharmaceuticals' EPI-743, according to the first-ever published report of disease reversal for the progressive and usually fatal neuromuscular disorder, which primarily is diagnosed in infants and children.