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Efficacy of KL1333 in Adult Patients With Primary Mitochondrial Disease (FALCON)

• Source: An Interventional, Randomised, Double-blind, Parallel-group, Placebo-controlled, Flexible-dose, Adaptive Study of the Efficacy of KL1333 in Adult Patients With Primary

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A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease

• Source: An Open-Label, Safety Study for Previously Treated Vatiquinone (PTC743) Subjects With Inherited Mitochondrial Disease

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Study to Evaluate Efficacy and Safety of Elamipretide in Subjects With Primary Mitochondrial Disease From Nuclear DNA Mutations (nPMD) (NuPower)

• Source: A Phase 3 Randomized, Double-Blind, Parallel-Group, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Daily Subcutaneous Injections of Elamipretide in Subjects With Primary

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Acute Infection in Mitochondrial Disease: Metabolism, Infection and Immunity

• Source: Acute Infection in Mitochondrial Disease: An Observational Prospective Natural History Study of Metabolism, Infection and Immunity

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Promoting Resilience in Stress Management (PRISM) and Clinical-focused Narrative (CFN) Pilot in Adults With Primary Mitochondrial Disease (PMD).

• Authors : North American Mitochondrial Disease Consortium (NAMDC); National Institute of Neurological Disorders and Stroke (NINDS)

• Source: Harnessing Resilience in Adults With Primary Mitochondrial Disease: A Pilot Study Investigating the Feasibility of the Promoting

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Expanded Access Program for R007 (Probucol) Tablets in Adults With Mitochondrial Disease and Chronic Kidney Disease

• Authors : Children's Hospital of Philadelphia

• Source: Expanded Access Treatment Protocol for R007 (Probucol) Tablets in Adults With Genetically-Confirmed Mitochondrial Disease and Chronic

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Neurometabolic Profile of Individuals With Primary Mitochondrial Disease

• Source: Neurometabolic Profile of Individuals With Primary Mitochondrial Disease

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Applying pGz in Mitochondrial Disease

• Authors : United States Department of Defense

• Source: The Utility of pGz in Primary Mitochondrial DisordersSackner MA, Lopez JR, Banderas V, Adams JA. Can Physical Activity While Sedentary Produce Health Benefits? A

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Open-Label, Dose-Escalating Study Assessing Safety, Tolerability, Efficacy, of RP103 in Mitochondrial Disease (MITO-001)

• Source: An Open-Label, Dose-Escalating Study to Assess the Safety, Tolerability, Efficacy, Pharmacokinetics and Pharmacodynamics of Cysteamine Bitartrate Delayed-release Capsules (RP103) for Treatment of

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CrCest Study in Primary Mitochondrial Disease

• Source: Magnetic Resonance Imaging (MRI) Muscle Phenotyping in Mitochondrial Disease

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