A Study to Evaluate Vitamin B3 Derivative to Treat Mitochondrial Myopathy

Eligibility Criteria

• * Biochemically and/or genetically confirmed or confirmed primary mitochondrial myopathy based on published diagnostic criteria.
• * Biochemically confirmed mitochondrial disorder would mean that the patient meets clinical criteria and has either biopsy or biochemical testing that supports the diagnosis.
• * Confirmed mitochondrial disorder means that the patient meets published clinical criteria for the diagnosis and has also had confirmatory genetic testing for the disorder type.
• * Agreed to avoid vitamin supplementation or nutritional products with vitamin B3 forms 14 days prior to the enrollment and during the study in order to not exceed 200 mg/day of vitamin B3 derivatives intake.
• * Female of childbearing potential agreed to use effective contraception throughout the study.
• * Written, informed consent to participate in the study.
• * Unwilling to comply with the follow-up schedule.
• * Inability or refusal to give informed consent by the patient or a legally authorized representative.
• * Known pregnancy or breastfeeding.
• * Concurrent participation in another investigational drug study or within washout period of treatment.
• * Presence of other medical symptoms or condition, which may interfere with interpretation of outcome measures as determined by the study PI.