D Moreno-Martinez, P Aguiar, C Auray-Blais, M Beck, D G Bichet, A Burlina, D Cole, P Elliott, U Feldt-Rasmussen, S Feriozzi, J Fletcher, R Giugliani, A Jovanovic, C Kampmann, M Langeveld, O Lidove, A Linhart, M Mauer, J C Moon, A Muir, A Nowak, J P Oliveira, A Ortiz, G Pintos-Morell, J Politei, P Rozenfeld, R Schiffmann, E Svarstad, A S Talbot, M Thomas, C Tøndel, D Warnock, M L West, D A Hughes
BACKGROUND: Recent years have witnessed a considerable increase in clinical trials of new investigational agents for Fabry disease (FD). Several trials investigating different agents are currently in progress; however, lack of standardisation results in challenges to interpretation and comparison. To facilitate the standardisation of investigational programs, we have developed a common framework for future clinical trials in FD. METHODS AND FINDINGS: A broad consensus regarding clinical outcomes and ways to measure them was obtained via the Delphi methodology...
April 2021: Molecular Genetics and Metabolism