Progression characteristics of the European Friedreich's Ataxia Consortium for Translational Studies (EFACTS): a 4-year cohort study

Prof Kathrin Reetz, MD, Imis Dogan, PhD, Prof Ralf-Dieter Hilgers, PhD, Prof Paola Giunti, MD, Michael H Parkinson, MBBS, Caterina Mariotti, MD, Lorenzo Nanetti, MD, Prof Alexandra Durr, MD, Claire Ewenczyk, MD, Sylvia Boesch, MDWolfgang Nachbauer, MD, Thomas Klopstock, MD,, Claudia Stendel, MD, Francisco Javier Rodríguez de Rivera Garrido, MD, Christian Rummey, PhD, Prof Ludger Schöls, MD, Stefanie N Hayer, PhD Prof Thomas Klockgether, MD, Ilaria Giordano, MD, Claire Didszun, PhD, Myriam Rai, PhD, Prof Massimo Pandolfo, MD, Prof Jörg B Schulz on behalf of theEFACTS study group; The Lancet Neurology, Published:March 23, 2021 DOI:10.1016/S1474-4422(21)00027-2 

The European Friedreich's Ataxia Consortium for Translational Studies (EFACTS) investigates the natural history of Friedreich's ataxia. We aimed to assess progression characteristics and to identify patient groups with differential progression rates based on longitudinal 4-year data to inform upcoming clinical trials in Friedreich's ataxia.

Progression characteristics of the European Friedreich's Ataxia Consortium for Translational Studies (EFACTS): a 4-year cohort study