ABSTRACT
Abstract Mitochondrial dysfunction results in the production of an abnormally high amount of reactive oxygen and nitrogen species, which results in redox imbalance and glutathione deficiency. Therapeutics such as EPI-743 (?-tocotrienol quinone) and RP103 (cysteamine bitartrate) have the theoretical potential to improve redox imbalance by increasing intracellular glutathione and are currently under investigation in multiple clinical trials. This review provides an update on the use of these compounds in clinical trials related to primary and secondary mitochondrial disorders. These clinical trials have not only provided hope to affected patients and their families and caregivers, but also will serve as important stepping stones for further studies as our understanding of mitochondrial disease pathogenesis continues to improve.
ABSTRACT
Leber's hereditary optic neuropathy (LHON) is an inherited mitochondrial disorder characterized by bilateral progressive vision loss.Current management includes therapies directed at enhancing mitochondrial function and preventing oxidative damage.This article reviews the progress of treatments from mitochondria cocktail,idebenone,gene therapy,EPI-743,brimonidine,traditional Chinese medicine and physical therapy,providing a new insight in the treatments of LHON.