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Inherited Mitochondrial Disease
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Inherited Mitochondrial Disease trials may be worth asking aboutClinical trial pipeline · Data from ClinicalTrials.gov
See which Inherited Mitochondrial Disease trials may be worth asking aboutNorth America
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Trials may have sites in multiple countries. Filtering shows any trial with at least one site in the selected country.
The KHENERFIN study aims to determine whether the study medicine, sonlicromanol, is able to reduce symptoms of fatigue and the impact of fatigue on daily life,…
This study aims to gather safety data and determine the optimal dosing regimen for PYC-001 in participants with confirmed OPA1 mutation-associated ADOA. Approxi…
This study will evaluate the use of autologous bone marrow derived stem cells (BMSC) for the treatment of retinal and optic nerve damage or disease.
The purpose of this double-masked, randomized, placebo-controlled, paired-eye study is to evaluate the efficacy, safety and tolerability of Sepofarsen in subjec…
The Metabolism, Infection and Immunity (MINI) Study is a longitudinal natural history study at the National Institutes of Health (NIH) that aims to define the r…
This is a phase 1b, first in-human, open-label, dose-finding study investigating the safety and tolerability of SGT-212 in participants with Friedreich's ataxia…
Work participation is essential for quality of life, providing purpose, social interaction, financial security, and shaping social status. Work participation is…
The goal of this study is to obtain gamma-aminobutyric acid (GABA) and glutathione (GSH) assessment derived from magnetic resonance spectroscopy (MRS), to be us…