Therapy for mitochondrial disorders: Little proof, high research activity, some promise

SummaryMitochondrial disorders are a common group of metabolic diseases, the largest subgroup being the respiratory chain deficiencies. The most severe forms of mitochondrial dysfunction manifest in the neonatal period, rendering this group of patients the most challenging for therapy development. Heterogeneity of molecular backgrounds in the whole mitochondrial disease group has hindered therapy trials, but promising results are being gained from studies on animal models. Here I review strategies that have been tested or that can be proposed to be feasible as intervention. Many of these strategies aim to slow down the disease progression or are palliative in nature. However, currently very little evidence for any kind of therapeutic tools is available from double-blind controlled studies.