Mitochondrial Diseases
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CLINICAL_TRIALS trial_result · 2026-04-22Clinical Trial Update: Effect Of The System In The Enriched Fat Metabolism Of The Child Reached Mitochondrial Cytopathy [NA]
A completed clinical trial evaluating a system for enriched fat metabolism in children with mitochondrial diseases.
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CLINICAL_TRIALS trial_result · 2026-04-20Clinical Trial Update: Digital Health Technology for People With Mitochondrial Disease [NA]
The clinical trial update indicates that the study evaluating digital health technology for people with mitochondrial diseases has completed…
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CLINICAL_TRIALS research · 2026-04-20Clinical Trial Update: The Natural History of Mitochondrial Diseases
A clinical trial is recruiting to characterize the natural history of mitochondrial diseases.
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EMA approval · 2026-04-15EMA Authorised: Kygevvi (doxecitine;Doxribtimine) — UCB Pharma
The EMA authorized Kygevvi (doxecitine; Doxribtimine) from UCB Pharma for mitochondrial diseases, with orphan medicine designation.
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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 · 2026-03-05Clinical Trial Update: Exercise Training Effects on Muscle Function in Adults With Mitochondrial Myopathy [NA]
This clinical trial update indicates an ongoing study evaluating the effects of exercise training on muscle function in adults with mitochon…
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CLINICAL_TRIALS research · 2026-02-19Clinical Trial Update: Mitochondrial Substrate Utilization in the Diabetic Human Heart
A clinical trial is currently recruiting to study mitochondrial substrate utilization in the diabetic human heart, focusing on heart failure…
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CLINICAL_TRIALS research · 2026-02-18Clinical Trial Update: Validation of Oxygen Nanosensor in Mitochondrial Myopathy [Phase 1]
A Phase 1 clinical trial is underway to validate an oxygen nanosensor for use in mitochondrial myopathies and related mitochondrial diseases…
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CLINICAL_TRIALS research · 2026-02-04Clinical Trial Update: North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC)
The entry notes ongoing recruitment for the NAMDC North American Mitochondrial Disease Consortium Patient Registry and Biorepository to coll…
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CLINICAL_TRIALS research · 2026-01-29Clinical Trial Update: KHENERFIN Study: A Trial to Evaluate the Efficacy and Safety of Sonlicromanol in Primary Mitochondrial Diseases [Phase 3]
Khondrion's KHENERFIN study is a Phase 3 trial evaluating Sonlicromanol for mitochondrial diseases, currently recruiting.
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CLINICAL_TRIALS research · 2026-01-14Clinical Trial Update: CrCest Study in Primary Mitochondrial Disease
The CrCest Study for primary mitochondrial disease is currently enrolling by invitation, indicating an ongoing clinical research effort with…
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CLINICAL_TRIALS trial_result · 2025-11-28Clinical Trial Update: Reference Values of Intraepidermal Nerve Fiber Density in Children and Small Fiber Neuropathy in Neurodevelopmental Disorders
This clinical trial update provides reference values for intraepidermal nerve fiber density in children. It also investigates small fiber ne…
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CLINICAL_TRIALS research · 2025-11-10Clinical Trial Update: Applying pGz in Mitochondrial Disease [Phase 1]
A Phase 1 clinical trial conducted by Children’s Hospital of Philadelphia is recruiting to evaluate pGz in mitochondrial diseases, including…
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CLINICAL_TRIALS trial_result · 2025-10-24Clinical Trial Update: Study to Evaluate Efficacy and Safety of Elamipretide in Subjects With Primary Mitochondrial Disease From Nuclear DNA Mutations (nPMD) [Phase 3]
Phase 3 study evaluating the efficacy and safety of elamipretide in subjects with primary mitochondrial disease from nuclear DNA mutations h…
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CLINICAL_TRIALS research · 2025-10-15Clinical Trial Update: Longitudinal Study of Mitochondrial Hepatopathies
The longitudinal study of mitochondrial hepatopathies is currently suspended.
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CLINICAL_TRIALS other · 2025-10-01Clinical Trial Update: Collection of Blood and Skin Samples From Patients With Primary Mitochondrial Diseases and Healthy Volunteers
This clinical trial update notes ongoing recruitment for a study collecting blood and skin samples from patients with primary mitochondrial …
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CLINICAL_TRIALS research · 2025-07-20Clinical Trial Update: Mitochondrial Donation: An 18 Month Outcome Study.
An update on an ongoing 18-month outcome study of mitochondrial donation has been released. The trial is currently recruiting at Newcastle-u…
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CLINICAL_TRIALS research · 2025-06-22Clinical Trial Update: Mitochondrial Dysfunctions Driving Insulin Resistance
A completed clinical trial update from Rigshospitalet examining how mitochondrial dysfunction may drive insulin resistance in mitochondrial …
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CLINICAL_TRIALS research · 2025-06-22Clinical Trial Update: Evaluate the Safety and Therapeutic Effects of a Single Intravenous Infusion (IV) of Autologous CD34+ Cells Enriched With Allogenic Placenta-derived Mitochondria in Patients With a Diagnosis of Pearson Syndrome (PS) [Phase 2]
A Phase 2 clinical trial is evaluating the safety and potential therapeutic effects of a single IV infusion of autologous CD34+ cells enrich…
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CLINICAL_TRIALS trial_result · 2025-06-22Clinical Trial Update: Exercise-mediated Rescue of Mitochondrial Dysfunctions Driving Insulin Resistance [NA]
A completed clinical trial evaluated whether exercise can rescue mitochondrial dysfunction that drives insulin resistance.
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CLINICAL_TRIALS withdrawal · 2025-06-22Clinical Trial Update: A First in Human Study to Evaluate the Safety of Infusion of MNV-BM-PLC (Autologous CD34+ Cells Enriched With Placenta Derived Allogeneic Mitochondria) in Patients With Primary Mitochondrial Diseases Associated With Mitochondrial DNA Mutation or Deletion [Phase 1]
First-in-human Phase 1 trial evaluating the safety of infusing autologous CD34+ cells enriched with placenta-derived allogeneic mitochondria…
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CLINICAL_TRIALS research · 2025-05-31Clinical Trial Update: Deoxynucleosides Pyrimidines as Treatment for Mitochondrial Depletion Syndrome [Phase 2]
A Phase 2 clinical trial assessing deoxynucleosides pyrimidines for mitochondrial depletion syndrome is currently recruiting.
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CLINICAL_TRIALS trial_result · 2025-03-30Clinical Trial Update: Mitochondrial Diseases - Long-read Genome and Transcriptome Sequencing in Cases Unresolved After Short-read Genomics [NA]
A completed clinical trial evaluating long-read genome and transcriptome sequencing to resolve cases unresolved by short-read genomic method…
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CLINICAL_TRIALS research · 2025-02-28Clinical Trial Update: Combating the Diagnostic Impasse in Mitochondrial Diseases: a Transcriptomic Approach in Fibroblasts and Blood Cells [NA]
This clinical trial is exploring a transcriptomic approach to diagnose mitochondrial diseases using fibroblasts and blood cells. It is curre…
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CLINICAL_TRIALS research · 2025-02-25Clinical Trial Update: MITOMICS : a Multi-OMICS Approach for the Diagnosis of Mitochondrial Diseases
This entry announces a recruiting clinical trial (MITOMICS) that aims to diagnose mitochondrial diseases using a multi-omics approach. It is…