Inborn mitochondrial myopathy
MONDO:0009637Myopathy caused by mitochondrial abnormalities.
Also known as: mitochondrial myopathy
56 clinical trials for this condition and its sub-types, 18 tagged with Inborn mitochondrial myopathy itself.
Follow this condition to get notified about new trialsWhere it sits in the disease tree
Browse by category →Sub-types of Inborn mitochondrial myopathy
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Mitochondrial encephalomyopathy 3 trials · 15 incl. sub-types
2 sub-types
- MELAS syndrome 13 trials · 14 incl. sub-types Sub-types →
- MERRF syndrome 5 trials
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Progressive external ophthalmoplegia 4 trials · 8 incl. sub-types
3 sub-types
- Kearns-Sayre syndrome 5 trials
- Progressive external ophthalmoplegia with mitochondrial DNA deletions 0 trials · 2 incl. sub-types Sub-types →
- Autosomal recessive progressive external ophthalmoplegia 0 trials Sub-types →
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Barth syndrome 5 trials
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4 sub-types
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2 sub-types
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3 sub-types
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1 sub-type
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4 sub-types
- Cardioencephalomyopathy, fatal infantile, due to cytochrome c oxidase deficiency 1 0 trials
- Cardioencephalomyopathy, fatal infantile, due to cytochrome c oxidase deficiency 2 0 trials
- Cardioencephalomyopathy, fatal infantile, due to cytochrome c oxidase deficiency 3 0 trials
- Cardioencephalomyopathy, fatal infantile, due to cytochrome c oxidase deficiency 4 0 trials
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4 sub-types
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Mitochondrial myopathy with diabetes 0 trials
Most studied deeper sub-types
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Experimental drug aims to boost energy in rare genetic disorders
Disease control Not yet recruitingThis study tests an oral drug called glycerol tributyrate in 24 adults with MELAS or LHON-Plus, two rare mitochondrial diseases that cause severe symptoms like strokes and vision loss. The trial is open-label (everyone gets the drug) and uses each person as their own control over…
Phase 1/2 • Sponsor: George Washington University • Aim: Disease control
Last updated Jun 27, 2026 13:02 UTC
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Could your own stem cells fight this rare brain disorder?
Disease control Not yet recruitingThis study tests whether a person's own stem cells, processed and given by IV, can safely help with multiple system atrophy (MSA) — a rare, worsening brain disease that affects movement and automatic body functions like blood pressure. Fifty adults aged 35 to 65 will receive eith…
Phase 2 • Sponsor: Biocells Medical • Aim: Disease control
Last updated Jun 27, 2026 11:01 UTC
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New DNA test could end years of uncertainty for mitochondrial disease patients
Diagnosis Not yet recruitingThis pilot study aims to develop a new digital PCR technique to more accurately diagnose mitochondrial diseases. Researchers will test the method on blood, urine, saliva, and muscle fiber samples from 4 patients. If validated, the technique could be faster and cheaper than curren…
Sponsor: Centre Hospitalier Universitaire de Nice • Aim: Diagnosis
Last updated Jun 27, 2026 12:04 UTC