Mito research around the world

An exciting time for mitochondrial research

This is a very exciting time for research into mitochondrial disease. Growing awareness about the condition and improved diagnosis have resulted in more global research centres undertaking valuable work on potential drug treatments.

Scientists have linked mitochondrial dysfunction with other serious diseases including cancer, diabetes, Alzheimer's and stroke, making this an even more vital field of study.

While there have been several exciting breakthroughs in recent years, many studies are still a long way from clinical trials. The information provided here focuses on the most promising looking treatments, which we hope will be of benefit to patients in the near future.

Current research projects around the world:

Bezafibrate

In clinical trial.

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Acipimox

Has been approved for clinical trial.

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MT1621 (nucleoside therapy)

In clinical trial.

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Cysteamine Bitartrate (RP103)

Clinical trial discontinued.

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EPI-743

In clinical trial.

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Elamipretide (Bendavia, MTP-131)

In clinical trial.

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Genome Editing using CRISPR/Cas

In development.

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GS010 Gene Therapy

In clinical trial.

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Idebenone (Raxone)

In clinical trial.

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JOTROL

Clinical trial planned.

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KH-176

In clinical trial.

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KL-1333

In clinical trial.

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NV354

Pre-clinical research.

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Nucleoside Bypass Therapy & Mitochondrial DNA Depletion Syndromes

Pre-clinical research.

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Omaveloxone

In clinical trial.

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