Background and Objectives
GENETIC DISORDERS, MITOCHONDRIAL MYOPATHIES
Go to The Article Processing Charge was funded by Stealth Biotherapeutics.Coinvestigators are listed at This Null Hypothesis article is published as part of a collaborative effort between NeurologySubmitted and externally peer reviewed. The handling editor was Associate Editor Anthony Amato, MD, FAAN.Primary mitochondrial myopathies (PMMs) encompass a group of genetic disorders that impair mitochondrial oxidative phosphorylation, adversely affecting physical function, exercise capacity, and quality of life (QoL). Current PMM standards of care address symptoms, with limited clinical impact, constituting a significant therapeutic unmet need. We present data from MMPOWER-3, a pivotal, phase-3, randomized, double-blind, placebo-controlled clinical trial that evaluated the efficacy and safety of elamipretide in participants with genetically confirmed PMM.