The changing landscape of clinical trials for mitochondrial diseases: 2011 to present.

We reviewed the status of interventional clinical trials for primary mitochondrial diseases. Using national and international search engines, we found 48 randomized controlled trials (RCTs) registered as of May 15, 2019. Consilience between lay and professional mitochondrial disease communities to engage in RCTs has increased, as has progress in developing new disease and treatment biomarkers and potential therapies. The continued advancement of general knowledge of mitochondrial biology has fostered appreciation for the fundamental role mitochondria play in the etiopathology of other rare and common illnesses, emphasizing the therapeutic potential of mitochondrially-targeted small molecules for an increasing spectrum of human diseases.