Developing Clinical Biomarkers for Mitochondrial Disease for Therapeutic Trials

Recent advances in understanding the mechanisms of mitochondrial diseases have led to the development of new treatment approaches that show real promise in animal models. The field has also come together to assemble genetically defined cohorts of patients, ready for interventional studies. However, there are a limited number of clinically-relevant biomarkers available to assess disease progression before or during treatment. The aim of this retreat was to bring together experts to help break down the biomarker ‘road-block’ and lead to well-informed treatment trials.

Scientific programme committee