Joana Carvalho, PhD,  —

GenSight Biologics annnounced the topline results of a Phase 3 clinical trial for GS010, a gene therapy designed to improve vision acuity in patients with Leber hereditary optic neuropathy (LHON), a rare genetic mitochondrial disease that causes vision loss. The randomized, double-blind, placebo-controlled trial (NCT02652767), called RESCUE,…

Researchers found an enzyme called alternative oxidase (AOX) that is able to bypass part of the mitochondrial respiratory chain (MRC) and alleviate symptoms of mitochondrial cardiomyopathy in a mouse model of the disease. The study, “Alternative oxidase‐mediated respiration prevents lethal mitochondrial cardiomyopathy,” was published in EMBO Molecular…

GS010, a gene therapy developed by GenSight Biologics, improves visual acuity and quality of life in patients with Leber hereditary optic neuropathy (LHON), a rare genetic mitochondrial disease that causes vision loss, Phase 3 trial data shows. The findings are based on Week 72 data from the ongoing…

Women with mitochondrial disease or mitochondrial dysfunction are more susceptible to complications during pregnancy, and their newborns are more likely to have congenital defects, a retrospective study shows. The study, “Effects of mitochondrial disease/dysfunction on pregnancy: A retrospective study,” was published in Mitochondrion. Primary mitochondrial disease (MD) comprises several…