MMPOWER acute dose study in primary mitochondrial myopathy
Highest-dose group improved 64.5 m versus 20.4 m with placebo; nominal P=.053, with dose-response signal and adjusted analyses favoring high dose.
What this study tells us
Participants were assigned to comparison groups by chance, usually with placebo and/or blinding to reduce bias. This is a strong design for testing cause and effect, but the population, duration and endpoints still limit what can be concluded.
Primary mitochondrial myopathy
n=36
Highest-dose group improved 64.5 m versus 20.4 m with placebo; nominal P=.053, with dose-response signal and adjusted analyses favoring high dose.
Small exploratory study; short duration; nominal primary comparison did not cross conventional significance threshold.
Need help with terms such as randomized, placebo, endpoint or sample size? Open the glossary →
Key result
Highest-dose group improved 64.5 m versus 20.4 m with placebo; nominal P=.053, with dose-response signal and adjusted analyses favoring high dose.