Efficacy and Safety of Elamipretide in Individuals With Primary Mitochondrial Myopathy: The MMPOWER-3 Randomized Clinical Trial
Karaa A, Bertini E, Carelli V, et al. Neurology 2023;101:e238-e252.
What it found
Missed both main goals: no difference from placebo in 6-minute walk distance (-3.2 m) or fatigue score. Elamipretide was well tolerated, with mostly mild to moderate adverse events.
Limitations
A large, well-designed negative trial. A later post hoc analysis looked at genetic subgroups, which can only generate hypotheses.
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