Primary mitochondrial myopathy: Clinical features and outcome measures in 118 cases from Italy.

Vincenzo Montano Francesco Gruosso Valerio Carelli Giacomo Pietro Comi Massimiliano Mirabella Costanza Lamperti Tiziana Mongini Olimpia Musumeci Serenella Servidei Paola ToninAntonio ToscanoAngela ModeneseGuido PrimianoMaria Lucia ValentinoSara BortolaniSilvia MarchetMegi MeneriGraziana TavillaGabriele SicilianoMichelangelo Mancuso

Published in: Neurology. Genetics (2020)

We characterized a large cohort of PMM by evaluating baseline mitochondrial biomarkers and functional scales that represent potential outcome measures to monitor the efficacy of treatment in clinical trials; these outcome measures will be further reinvestigated longitudinally to define the natural history of PMM.

Keyphrases

clinical trial
oxidative stress
randomized controlled trial
combination therapy
risk assessment
human health
open label
study protocol
myasthenia gravis