Mito Research Around The World

It is a very exciting time for mitochondrial disease research. Greater awareness of the condition and improved diagnosis have resulted in an increasing number of global research centres undertaking valuable work on potential drug treatments. 

Whilst a lot of information is published in medical journals, many of these breakthroughs are still a number of years away from clinical trials so we have focused our information on the treatments that are looking the most promising and that we hope will be of benefit to patients in the near future.

EPI-743

In clinical trial.

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Elamipretide (Bendavia, MTP-131)

In clinical trial.

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Omaveloxone

In clinical trial.

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Bezafibrate

In clinical trial.

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Cysteamine Bitartrate (RP103)

In clinical trial.

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GS010 Gene Therapy

In clinical trial.

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KL-1333

Has been approved for clinical trial.

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KH-176

In clinical trial.

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Idebenone (Raxone)

In clinical trial.

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Nucleoside Bypass Therapy & Mitochondrial DNA Depletion Syndromes

Pre-clinical research.

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Genome Editing using CRISPR/Cas

In development.

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