News

GenSight Biologics released new preliminary findings from a Phase 1/2 clinical trial assessing the safety and tolerability of the company’s lead investigational gene therapy, GS010, in patients with Leber’s hereditary optic neuropathy (LHON), a maternally inherited mitochondrial disease causing irreversible vision loss. The data show significant improvement for those in earlier stages of the disease. GS010…

Boston, Massachusetts-based clinical-stage biopharma Stealth BioTherapeutics has released positive results from the MMPOWER Phase 2 trial, a double-blind, dose-ascending, placebo-controlled study investigating the safety, tolerability, and efficacy of its lead drug candidate elamipretide for treating primary mitochondrial myopathy (muscle weakness) in genetically confirmed mitochondrial disease patients. The trial evaluated…

Obesity and metabolic disorders in a woman can be passed to future generations, even if a child is fed a healthy diet, and — at least in mice, the subjects of this study — may be a predetermined outcome even before the offspring are conceived. The study, “Maternal Metabolic Syndrome…

The Wellcome Trust Centre for Mitochondrial Disease has published strong evidence supporting a new in vitro fertilization (IVF) procedure that allows for mitochondrial replacement — lowering the risk of babies being born with inherited mitochondrial mutations. The findings, reported in the journal Nature, represent a huge step forward for research into mitochondrial…

Injured nerve cells in mature mice were better able to repair themselves when researchers boosted the transport of mitochondria along neuronal axons. This finding, which suggests a new way of working to stimulate regrowth in human neurons damaged by injury or disease, is reported in the study “Facilitation of axon regeneration by enhancing mitochondrial…

The Pittsburgh, Penn. based United Mitochondrial Disease Foundation (UMDF) will celebrate its 20th anniversary at the Mitochondrial Medicine 2016 Symposium this week in Seattle, Wash., according to a press release provided to Mitochondrial Disease News. Currently recognized as the world’s foremost mitochondrial disease symposium, just 10 years ago the UMDF…

Stealth BioTherapeutics, a biopharma that focuses on drugs for mitochondrial dysfunction, recently announced the start of a Phase 2 clinical trial exploring elamipretide (previously known as Ocuvia) in the form of eye drops for the treatment of Leber’s Hereditary Optic Neuropathy. Stealth’s lead candidate, elamipretide, works by preserving energy processes…

Stealth BioTherapeutics recently reported positive data from its Phase 2 MMPOWER clinical trial, a double-blind, placebo-controlled study that evaluated the safety, tolerability, and efficacy of elamipretide (Bendavia) in patients with mitochondrial myopathy (MM). The MMPOWER study investigated Bendavia for the treatment of myopathy (muscle weakness) in patients with genetic mitochondrial diseases. These diseases…

Researchers at Nizam's Institute of Medical Sciences in India reported a case of adult onset Leigh’s disease that showed dramatic improvement following treatment. This case study suggests that late onset Leigh’s disease may be treatable.

Researchers recently tested the effects of the high-fat, low carbohydrate ketogenic diet on fibroblasts from patients with Leigh syndrome (LS), focusing on decanoic acid. The results suggest that this component had beneficial effects on mitochondrial function, decreasing reactive oxygen species (ROS) and improving mitochondrial respiration. Therefore, the effects of this…