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Article

Efficacy and Safety of Elamipretide in Individuals With Primary Mitochondrial Myopathy

2 Jun 2023

Abstract excerpt

Background and Objectives: Primary Mitochondrial Myopathies (PMMs) encompass a group of genetic disorders that impair mitochondrial oxidative phosphorylation, adversely impacting 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,...

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PMID: 37268435
DOI: 10.1212/wnl.0000000000207402

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