Evaluating the efficacy of vatiquinone in preclinical models of mitochondrial disease.
Ernst-Bernhard KayserYihan ChenMichael MulhollandVivian TruongKaterina JamesAllison HanafordSimon C JohnsonPublished in: Research square (2024)
(-/-) model. Our findings are consistent with recent press statements regarding clinical trial results and have implications for drug trial design and reporting in patients with rare diseases.