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Review
. 2020 Jan:50:51-57.
doi: 10.1016/j.mito.2019.10.010. Epub 2019 Oct 25.

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

Delia Khayat  1 , Tracie L Kurtz  1 , Peter W Stacpoole  2
Affiliations
Review

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

Delia Khayat et al. Mitochondrion. 2020 Jan.

Abstract

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.

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Figures

Fig. 1.
Fig. 1.
Evolution of interventional clinical trials for primary mitochondrial diseases, 2011- Present. Red: citations in original study using only PubMed through February 11, 2011; blue: citations using PubMed through May 15, 2019; yellow: citations using all citations listed above through May 15, 2019.
Fig. 2.
Fig. 2.
Geography of Randomized Controlled Trials in Mitochondrial Diseases, 02/11/2011 – 05/15/2019. Data obtained from the expanded search engine list described in the text.
See this image and copyright information in PMC

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