Randomized, clinical trial of RT001: Early signals of efficacy in Friedreich's ataxia

Theresa Zesiewicz MD, FAAN, Frederic Heerinckx MPharm, Robert De Jager MD, FACP, Omid Omidvar MD, Marcus Kilpatrick PhD, Jessica Shaw MPH, Mikhail S. Shchepinov PhD, Movement Disorders : Official Journal of the Movement Disorder Society, First published: 6 April 2018 doi:10.1002/mds.27353

RT001 was found to be safe and tolerable over 28 days, and improved peak workload. Further research into the effect of RT001 in Friedreich's ataxia is warranted.

Randomized, clinical trial of RT001: Early signals of efficacy in Friedreich's ataxia