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Article

Randomized dose-escalation trial of elamipretide in adults with primary mitochondrial myopathy

2 Mar 2018

Abstract excerpt

<h3>Objective</h3> To assess the safety and efficacy of elamipretide, an aromatic-cationic tetrapeptide that readily penetrates cell membranes and transiently localizes to the inner mitochondrial membrane where it associates with cardiolipin, in adults with primary mitochondrial myopathy (PMM). <h3>Methods</h3> A Study Investigating the Safety, Tolerability, and Efficacy of MTP-131 for the Treatment of...

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PMID: 29500292
DOI: 10.1212/wnl.0000000000005255

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