This study, “Development of the Mitochondrial Video Assessment (MVA) as an Outcome Measure for the Evaluation of Patients with Mitochondrial Myopathies,” hopes to develop sensitive, reliable outcome measures that will provide decisive information on treatment efficacy using outcome variables that are most important and impactful to PolG and PMM patients. The study will employ remote video capture to assess subtle shifts in movement patterns to measure change in function over time, allowing patients to participate in clinical trials without the added stressors of traveling to a clinic site. Patients will be asked to record themselves doing disease-specialized tasks that measures things like ataxia and gait, which will then be professionally graded by a physical therapist, showing progress or regression during the study.
Requirements: Genetically confirmed PMM or POLG patients age 16+
To enroll or learn more, reach out to UMDF’s science team.