Mitochondrial Disease
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS research · 2026-04-23Clinical Trial Update: Transgenerational Metabolic-Immune Biomarkers of Neurological and Neurodevelopmental Disorders
Recruitment is ongoing for a study that investigates transgenerational metabolic-immune biomarkers in autism spectrum disorder and related m…
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CLINICAL_TRIALS research · 2026-04-20Clinical Trial Update: The NIH MINI Study: Metabolism, Infection, and Immunity in Inborn Errors of Metabolism
The NIH MINI study is currently recruiting to study metabolism, infection, and immunity in people with inborn errors of metabolism.
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CLINICAL_TRIALS research · 2026-04-15Clinical Trial Update: Efficacy of KL1333 in Adult Patients With Primary Mitochondrial Disease [Phase 2]
A Phase 2 clinical trial evaluating KL1333 in adults with primary mitochondrial disease is currently recruiting. Sponsor Abliva AB.
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CLINICAL_TRIALS withdrawal · 2026-04-09Clinical Trial Update: Vatiquinone Expanded Access Protocol
The expanded access protocol for vatiquinone in mitochondrial disease is no longer available.
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CLINICAL_TRIALS trial_result · 2026-04-07Clinical Trial Update: Open Label Extension to Assess Long Term Safety and Efficacy of KL1333 in Patients With Primary Mitochondrial Disease [Phase 2]
Open-label extension study to evaluate long-term safety and efficacy of KL1333 in patients with primary mitochondrial disease; Phase 2, not …
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CLINICAL_TRIALS research · 2026-04-06Clinical Trial Update: Development of a New Technique for Quantifying Mitochondrial DNA in Single Muscle Fibers [NA]
The document reports development of a new technique to quantify mitochondrial DNA in single muscle fibers, relevant to mitochondrial disease…
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CLINICAL_TRIALS trial_result · 2026-03-25Clinical Trial Update: A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy [Phase 2, Phase 3]
A Phase 2/3 study evaluating Vatiquinone for mitochondrial disease in participants with refractory epilepsy has been terminated, and results…
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CLINICAL_TRIALS research · 2026-02-17Clinical Trial Update: Validation of Nanosensor Oxygen Measurement [Phase 1]
This is an update on a Phase 1 clinical trial to validate a nanosensor-based oxygen measurement method. The study, sponsored by Children's H…
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CLINICAL_TRIALS research · 2026-02-05Clinical Trial Update: Neurometabolic Profile of Individuals With Primary Mitochondrial Disease
ClinicalTrials.gov study is actively recruiting to characterize the neurometabolic profile of individuals with primary mitochondrial disease…
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CLINICAL_TRIALS research · 2026-01-23Clinical Trial Update: Study of N-acetylcysteine in the Treatment of Patients With the m.3243A>G Mutation and Low Brain Glutathione Levels [Phase 1]
Phase 1 clinical trial update evaluating N-acetylcysteine for patients with the m.3243A>G mitochondrial DNA mutation and low brain glutathio…
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CLINICAL_TRIALS trial_result · 2026-01-22Clinical Trial Update: OMT-28 in Patients With Primary Mitochondrial Disease (PMD) (PMD-OPTION) [Phase 2]
Phase 2 clinical trial of OMT-28 in Primary Mitochondrial Disease has completed.
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CLINICAL_TRIALS research · 2026-01-22Clinical Trial Update: Mitochondrial Dysfunction in Autism Spectrum Disorder
Clinical trial update indicating the status of a study on mitochondrial dysfunction in autism spectrum disorder, currently active but not re…
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CLINICAL_TRIALS research · 2025-12-18Clinical Trial Update: Pilot Study of the Efficacy of Nicotinamide (Vitamin B3) in Leber's Hereditary Optic Neuropathy [Phase 1]
Phase 1 pilot trial evaluating nicotinamide (vitamin B3) for efficacy in Leber hereditary optic neuropathy; study status is not yet recruiti…
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CLINICAL_TRIALS research · 2025-10-02Clinical Trial Update: The Impact of Mitochondrial Dysfunction on Human Bone Cell Metabolism and Remodelling [NA]
Clinical trial update describing an ongoing study on how mitochondrial dysfunction affects human bone cell metabolism and remodeling, conduc…
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CLINICAL_TRIALS research · 2025-09-10Clinical Trial Update: Natural History Study of Mitochondrial Myopathy
This clinical trial update describes a natural history study of mitochondrial myopathy that is currently recruiting participants.
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CLINICAL_TRIALS research · 2025-08-01Clinical Trial Update: Acute Infection in Mitochondrial Disease: Metabolism, Infection and Immunity
A National Human Genome Research Institute-sponsored clinical trial on acute infection in mitochondrial disease, examining metabolism, infec…
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CLINICAL_TRIALS trial_result · 2025-06-22Clinical Trial Update: Human Mitochondrial Stress-driven Obesity Resistance
A completed clinical trial investigating human mitochondrial stress-driven obesity resistance.
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CLINICAL_TRIALS research · 2025-05-29Clinical Trial Update: Enhancement of Quality of Work And Life [NA]
ClinicalTrials.gov update for the study 'Enhancement of Quality of Work And Life' sponsored by Radboud University Medical Center, currently …
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CLINICAL_TRIALS research · 2025-04-17Clinical Trial Update: Expanded Access Program for R007 (Probucol) Tablets in Adults With Mitochondrial Disease and Chronic Kidney Disease
RiboNova Inc. has announced an expanded access program for R007 (Probucol) tablets in adults with mitochondrial disease and chronic kidney d…
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CLINICAL_TRIALS trial_result · 2025-04-15Clinical Trial Update: Mitochondrial Stress, Brain Imaging, and Epigenetics [NA]
Completed clinical trial investigating mitochondrial stress, brain imaging, and epigenetics in mitochondrial disease. Results have been post…
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CLINICAL_TRIALS research · 2025-02-14Clinical Trial Update: An Intermediate Size Expanded Access Protocol of Elamipretide
The update describes an intermediate-size expanded access protocol for elamipretide in patients with mitochondrial diseases, including Barth…
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CLINICAL_TRIALS trial_result · 2024-08-29Clinical Trial Update: Magnetic Resonance Imaging (MRI) Muscle Phenotyping in Mitochondrial Disease
This clinical trial update reports the completion of a study using MRI muscle phenotyping to characterize mitochondrial disease at the Unive…
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CLINICAL_TRIALS trial_result · 2024-07-17Clinical Trial Update: Anesthesia in Patients With Mitochondrial Disease [NA]
A clinical trial studying anesthesia in patients with mitochondrial disease was terminated, and its results have been posted on ClinicalTria…
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CLINICAL_TRIALS trial_result · 2021-12-17Clinical Trial Update: Open-Label Extension Trial to Characterize the Long-term Safety and Tolerability of Elamipretide in Subjects With Genetically Confirmed Primary Mitochondrial Myopathy (PMM) [Phase 2]
Open-label extension study terminated; aims to characterize long-term safety and tolerability of elamipretide in genetically confirmed prima…
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CLINICAL_TRIALS trial_result · 2021-10-07Clinical Trial Update: Assessment of Small Fiber Neuropathy in Rare Diseases Using Sudoscan [NA]
A completed clinical trial assessing small fiber neuropathy in Fabry disease and related conditions using Sudoscan; results posted in 2021.