Evaluating the efficacy of vatiquinone in preclinical models of mitochondrial disease.

ABSTRACT:

Background

Genetic mitochondrial diseases are a major challenge in modern medicine, impacting around 1:4,000 individuals. Leigh syndrome is the most common pediatric presentation of mitochondrial disease. There are currently no effective clinical treatments for mitochondrial disease. In humans, patients are often treated with antioxidants, vitamins, and strategies targeting energetics. The vitamin-E related compound vatiquinone (EPI-743, α-tocotrienol quinone) has been the subject of at least 19 clinical trials in the US since 2012, but the effects of vatiquinone on an animal model of mitochondrial disease have not yet been reported. Here, assessed the impact of vatiquinone on disease progression and in two animal models of mitochondrial disease.

Methods

The efficacy of vati

SUBMITTER: Kayser EB 

PROVIDER: S-EPMC11177993 | biostudies-literature | 2024 Jun

REPOSITORIES: biostudies-literature